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This publication is available at https://www.gov.uk/government/publications/orphan-registered-medicinal-products/orphan-register
Orphan registered products are listed in alphabetical order by trade name.
The list may be searched by pressing “CTRL+F” and entering a key word or phrase.
Where you are instructed to see a numbered section for more information, you can do so in the Summary of Product Characteristics (SmPC) for each product.
Orphan condition: Paroxysmal nocturnal haemoglobinuria
Authorised orphan indication: ASPAVELI is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months.
Orphan market exclusivity expiry date: 24 February 2032
GB Orphan Designation Number: PLGB 30941/0022/OD1
Orphan condition: Sickle cell disease
Adakveo is indicated for the prevention of recurrent vaso-occlusive crises (VOCs) in sickle cell disease patients aged 16 years and older. It can be given as an add on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.
Orphan market exclusivity date: 29 Oct 2030
GB Orphan Designation Number: PLGB 00101/1191/OD1
Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E
Orphan condition: Systemic anaplastic large cell lymphoma.
ADCETRIS in combination with cyclophosphamide, doxorubicin and prednisone (CHP) is indicated for adult patients with previously untreated systemic anaplastic large cell lymphoma (sALCL).
ADCETRIS is indicated for the treatment of adult patients with relapsed or refractory sALCL.
Orphan market exclusivity expiry date: 30 Oct 2024
GB Orphan designation number: PLGB 16189/0093/OD1
Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E
ADCETRIS is indicated for adult patients with previously untreated CD30+ Stage IV Hodgkin lymphoma (HL) in combination with doxorubicin, vinblastine and dacarbazine (AVD).
ADCETRIS is indicated for the treatment of adult patients with CD30+ HL at increased risk of relapse or progression following autologous stem cell transplant (ASCT).
ADCETRIS is indicated for the treatment of adult patients with relapsed or refractory CD30+ Hodgkin lymphoma (HL):
Orphan market exclusivity expiry date: 30 Oct 2024
GB Orphan designation number: PLGB 16189/0093/OD2
Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E
Orphan condition: Cutaneous T cell lymphoma
ADCETRIS is indicated for the treatment of adult patients with CD30+ cutaneous T cell lymphoma (CTCL) after at least 1 prior systemic therapy
Orphan market exclusivity expiry date: 19 Dec 2027
GB Orphan designation number: PLGB 16189/0093/OD3
Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate
Orphan condition: Chronic thromboembolic pulmonary hypertension (CTEPH)
Adempas is indicated for the treatment of adult patients with WHO Functional Class (FC) II to III with
Adempas, as monotherapy or in combination with endothelin receptor antagonists, is indicated for the treatment of adult patients with pulmonary arterial hypertension (PAH) with WHO Functional Class (FC) II to III to improve exercise capacity.
Efficacy has been shown in a PAH population including aetiologies of idiopathic or heritable PAH or PAH associated with connective tissue disease.
Orphan market exclusivity expiry date: 31 Mar 2024
GB Orphan designation number: PLGB 00010/0670 – 0674/OD1
Active substance: Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue
Alofisel is indicated for the treatment of complex perianal fistulas in adult patients with non-active/mildly active luminal Crohn’s disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy. Alofisel should be used after conditioning of fistulas.
Orphan market exclusivity expiry date: 27 Mar 2028
GB Orphan designation number: PLGB 16189/0094/OD1
Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1
Orphan condition: Haemophilia B (congenital factor IX deficiency)
Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency).
ALPROLIX can be used for all age groups.
Orphan market exclusivity expiry date: 13 May 2026
GB Orphan designation number: PLGB 30941/0003 – 0007/OD1
AMGLIDIA is indicated for the treatment of neonatal diabetes mellitus, for use in newborns, infants and children.
Sulphonylureas like AMGLIDIA have been shown to be effective in patients with mutations in the genes coding for the β-cell ATP-sensitive potassium channel and chromosome 6q24-related transient neonatal diabetes mellitus.
Orphan market exclusivity expiry date: 28 May 2028
GB Orphan designation number: PLGB 50687/0001 – 0004/OD1
Orphan condition: Non-tuberculous mycobacterial lung disease
ARIKAYCE liposomal is indicated for the treatment of non-tuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium Complex (MAC) in adults with limited treatment options who do not have cystic fibrosis.
Orphan market exclusivity expiry date: 28 Oct 2030
Orphan designation number: PLGB 47434/0001/OD1
(S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine
Orphan condition: Gastrointestinal stromal tumours
AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation
Orphan market exclusivity expiry date: 25 Sept 2030
Orphan designation number: PLGB 52115/0001 – 0003/0D1
Orphan condition: B-cell acute lymphoblastic leukaemia
BESPONSA is indicated as monotherapy for the treatment of adults with relapsed or refractory CD22-positive B cell precursor acute lymphoblastic leukaemia (ALL). Adult patients with Philadelphia chromosome positive (Ph+) relapsed or refractory B cell precursor ALL should have failed treatment with at least 1 tyrosine kinase inhibitor (TKI).
Orphan market exclusivity expiry date: 3 Jul 2027
GB Orphan designation number: PLGB 00057/1546/OD1
Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F
BLENREP is indicated as monotherapy for the treatment of multiple myeloma in adult patients, who have received at least four prior therapies and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy
Orphan market exclusivity expiry date: 26 Aug 2030
Orphan designation number: PLGB 19494/0296/OD1
Orphan condition: Acute lymphoblastic leukaemia
BLINCYTO is indicated as monotherapy for the treatment of adults with Philadelphia chromosome negative CD19 positive relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL).
BLINCYTO is indicated as monotherapy for the treatment of adults with Philadelphia chromosome negative CD19 positive B-precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%.
BLINCYTO is indicated as monotherapy for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation.
Orphan market exclusivity expiry date: 25 Nov 2025
GB Orphan designation number: PLGB 13832/0018/OD1
Orphan condition: Neuronal ceroid lipofuscinosis type 2 disease
Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Orphan market exclusivity expiry date: 1 Jun 2027
GB Orphan designation number: PLGB 45814/0001/OD1
Bronchitol is indicated for the treatment of cystic fibrosis (CF) in adults aged 18 years and above as an add-on therapy to best standard of care.
Orphan market exclusivity expiry date: 18 Apr 2022
GB Orphan designation number: PLGB 27944/0002/OD1
Orphan condition: Progressive familial intrahepatic cholestasis
Authorised orphan indication: Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older.
Orphan market exclusivity expiry date: 04 Aug 2031
GB Orphan designation number: PLGB 36216/0001-0004/OD1
Nanobody directed towards the human A1 domain of von Willebrand factor
Orphan condition: Thrombotic thrombocytopenic purpura
Cablivi is indicated for the treatment of adults and adolescents of 12 years of age and older weighing at least 40 kg experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression.
Orphan market exclusivity expiry date: 4 Sep 2030
GB Orphan designation number: PLGB 04425/0888/OD1
(1R, 2R)-Octanoic acid [2-(2’,3’-dihydro-benzo [1,4] dioxin-6’-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt
Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs).
Orphan market exclusivity expiry date: 21 Jan 2025
GB Orphan designation number: PLGB 04425/0763/OD1
Orphan condition: Inborn errors of primary bile acid synthesis
Chenodeoxycholic acid is indicated for the treatment of inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis (CTX)) in infants, children and adolescents aged 1 month to 18 years and adults
Orphan market exclusivity expiry date: 12 Apr 2027
GB Orphan designation number: PLGB 44975/0001/OD1
Active substance: Human coagulation factor X
Orphan condition: Hereditary factor X deficiency
Coagadex is indicated for the treatment and prophylaxis of bleeding episodes and for perioperative management in patients with hereditary factor X deficiency.
Coagadex is indicated in all age groups.
Orphan market exclusivity expiry date: 18 Mar 2028
GB Orphan designation number: PLGB 08801/0059 – 0060/OD1
Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt
Orphan condition: Medullary thyroid carcinoma
COMETRIQ is indicated for the treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma.
For patients in whom rearranged during transfection (RET) mutation status is not known or is negative, a possible lower benefit should be taken into account before individual treatment decision.
Orphan market exclusivity expiry date: 26 Mar 2024
GB Orphan designation number: PLGB 28247/0004 – 0005/OD1
Treatment of mucormycosis in patients for whom amphotericin B is inappropriate
Consideration should be given to official guidance on the appropriate use of antifungal agents.
Orphan market exclusivity expiry date: 19 Oct 2025
GB Orphan designation number: PLGB 32205/0005 – 0006/OD1
Consideration should be given to official guidance on the appropriate use of antifungal agents.
Orphan market exclusivity expiry date: 19 Oct 2025
GB Orphan designation number: PLGB 32205/0005 – 0006/OD2
Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23
CRYSVITA is indicated for the treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons
Orphan market exclusivity expiry date: 21 Feb 2028
GB Orphan designation number: PLGB 50262/0001/OD1
Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children from 2 years of age with cystinosis.
Orphan market exclusivity expiry date: 23 Jan 2027
GB Orphan designation number: PLGB 15266/0021/OD1
Orphan condition: Acute myeloid leukaemia
Dacogen is indicated for the treatment of adult patients with newly diagnosed de novo or secondary acute myeloid leukaemia (AML), according to the World Health Organisation (WHO) classification, who are not candidates for standard induction chemotherapy.
Orphan market exclusivity expiry date: 24 Sep 2024
GB Orphan designation number: PLGB 00242/0662/OD1
Orphan condition: Plasma cell myeloma
Orphan market exclusivity expiry date: 24 May 2026
GB Orphan designation number: PLGB 00242/0676 – 0677/OD1
Orphan condition: Systemic light chain (AL) amyloidosis
Authorised orphan indication: Darzalex is indicated in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic light chain (AL) amyloidosis
Orphan market exclusivity expiry date: 11 October 2031
GB Orphan designation number: PLGB 00242/0677/OD2
Orphan condition: Acute myeloid leukaemia
Daurismo is indicated, in combination with low-dose cytarabine, for the treatment of newly diagnosed de novo or secondary acute myeloid leukaemia (AML) in adult patients who are not candidates for standard induction chemotherapy.
Orphan market exclusivity expiry date: 29 Jun 2030
GB Orphan designation number: PLGB 00057/1687 – 1688/OD1
Orphan condition: Hepatic veno-occlusive disease
Treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy.
It is indicated in adults and in adolescents, children and infants over 1 month of age.
Orphan market exclusivity expiry date: 22 Oct 2023
GB Orphan designation number: PLGB 31626/0005/OD1
(S)-2-nitro-6-(4-(trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b][1,3]oxazine
Dovprela is indicated in combination with bedaquiline and linezolid, in adults, for the treatment of pulmonary extensively drug resistant (XDR), or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB).
Orphan market exclusivity expiry date: 4 Aug 2030
GB Orphan designation number: PLGB 46302/0234/OD1
Epidyolex is indicated for use as adjunctive therapy of seizures associated with Lennox Gastaut syndrome (LGS), in conjunction with clobazam, for patients 2 years of age and older.
Orphan market exclusivity expiry date: 23 Sept 2029
GB Orphan designation number: PLGB 36772/0001/OD1
Epidyolex is indicated for use as adjunctive therapy of seizures associated with Dravet syndrome (DS), in conjunction with clobazam, for patients 2 years of age and older.
Orphan market exclusivity expiry date: 23 Sept 2029
GB Orphan designation number: PLGB 36772/0001/OD2
Orphan condition: Tuberous sclerosis complex
Authorised orphan indication: Epidyolex is indicated for use as adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC) for patients 2 years of age and older.
Orphan market exclusivity expiry date: 05 Aug 2031
GB Orphan designation number: PLGB 36772/0001/OD3
Orphan condition: Spinal Muscular Atrophy
Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
Orphan market exclusivity expiry date: 19 May 2031
GB Orphan designation number: PLGB 00031/0920/OD1
Orphan condition: Treatment of multiple myeloma
Farydak, in combination with bortezomib and dexamethasone, is indicated for the treatment of adult patients with relapsed and/or refractory multiple myeloma who have received at least two prior regimens including bortezomib and an immunomodulatory agent.
Orphan market exclusivity expiry date: 1 Sep 2025
GB Orphan designation number: PLGB 53958/0007 – 0009/OD1
Fintepla is indicated for the treatment of seizures associated with Dravet syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older.
Orphan market exclusivity expiry date: 21 Dec 2030
GB Orphan designation number: PLGB 45831/0001/OD1
Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation (see the tables in section 5.1).
Orphan market exclusivity expiry date: 31 May 2026
GB Orphan designation number: PLGB 25823/0002/OD1
Orphan condition: Chronic lymphocytic leukaemia (CLL)
Gazyvaro in combination with chlorambucil is indicated for the treatment of adult patients with previously untreated CLL and with comorbidities making them unsuitable for full-dose fludarabine based therapy (see section 5.1).
Orphan market exclusivity expiry date: 24 Jul 2024
GB Orphan designation number: PLGB 00031/0856/OD1
Orphan condition: Follicular lymphoma (FL)
Gazyvaro in combination with chemotherapy, followed by Gazyvaro maintenance therapy in patients achieving a response, is indicated for the treatment of patients with previously untreated advanced FL (see section 5.1)
Gazyvaro in combination with bendamustine followed by Gazyvaro maintenance is indicated for the treatment of patients with FL who did not respond or who progressed during or up to 6 months after treatment with rituximab or a rituximab-containing regimen.
Orphan market exclusivity expiry date: 15 Jun 2026
GB Orphan designation number: PLGB 00031/0856/OD2
Orphan condition: Acute hepatic porphyria
Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA covalently linked to a ligand containing three N-acetylgalactosamine residues
Treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older
Orphan market exclusivity expiry date: 4 Mar 2030
GB Orphan designation number: PLGB 43942/0001/OD1
Indicated for use as part of an appropriate combination regimen for multi-drug resistant tuberculosis in adults and paediatric patients from 28 days of age and older when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability.
Consideration should be given to official guidance on the appropriate use of antibacterial agents.
Orphan market exclusivity expiry date: 9 Apr 2024
GB Orphan designation number: PLGB 35068/0003/OD1
Orphan condition: Hepatitis delta virus infection
The treatment of chronic hepatitis delta virus (HDV) infection in plasma (or serum) HDV-RNA positive adult patients with compensated liver disease.
Orphan market exclusivity expiry date: 05 Aug 2030
GB Orphan designation number: PLGB 50662/0002/OD1
Orphan condition: Non-24-hour sleep-wake disorders in blind people with no light perception
HETLIOZ is indicated for the treatment of Non-24-Hour Sleep-Wake Disorder (Non-24) in totally blind adults.
Orphan market exclusivity expiry date: 7 Jul 2025
GB Orphan designation number: PLGB 43460/0001/OD1
Ex-vivo expanded autologous human corneal epithelium containing stem cells
Orphan condition: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Treatment of adult patients with moderate to severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns. A minimum of 1-2 mm2 of undamaged limbus is required for biopsy.
Orphan market exclusivity expiry date: 19 Feb 2025
GB Orphan designation number: PLGB 53298/0001/OD1
Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl]
Orphan condition: Acute lymphoblastic leukaemia
Iclusig is indicated in adult patients with
Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.
Orphan market exclusivity expiry date: 3 Jul 2023
GB Orphan designation number: PLGB 49635/0001 – 0003/OD1
Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl]
Orphan condition: Chronic myeloid leukaemia
Iclusig is indicated in adult patients with
Orphan market exclusivity expiry date: 3 Jul 2023
GB Orphan designation number: PLGB 49635/0001 – 0003/OD2
Recombinant IgG degrading enzyme of Streptococcus pyogenes
Orphan condition: Graft rejection following solid organ transplantation
Idefirix is indicated for desensitisation treatment of highly sensitised adult kidney transplant patients with positive crossmatch against an available deceased donor. The use of Idefirix should be reserved for patients unlikely to be transplanted under the available kidney allocation system including prioritisation programmes for highly sensitised patients.
Orphan market exclusivity expiry date: 1 Sep 2030
GB Orphan designation number: PLGB 46323/0002/OD1
Recombinant fusion protein linking human coagulation factor IX with human albumin
Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency).
IDELVION can be used for all age groups.
Orphan market exclusivity expiry date: 13 May 2026
GB Orphan designation number: PLGB 00231/0337 – 0340/OD1
Orphan condition: genetic obesity disorders caused by defects in melanocortin 4 receptor (MC4R) pathway, including POMC and LEPR deficiency obesity
Imcivree is used for the treatment of obesity and the control of hunger associated with genetically confirmed loss of function biallelic proopiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.
Orphan market exclusivity expiry date: 14 September 2031
GB Orphan Designation Number: PLGB 55587/0001/OD1
Imnovid in combination with dexamethasone is indicated in the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including both lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy
Orphan market exclusivity expiry date: 8 Aug 2023
GB Orphan designation number: PLGB 50412/0002 – 0005/OD1
Active substance: fedratinib dihydrochloride monohydrate
Inrebic is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis who are Janus Associated Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.
Orphan market exclusivity expiry date: 16 April 2031
GB Orphan designation number: PLGB 50412/0022/OD1
Orphan condition: Cushing’s syndrome
Isturisa is indicated for the treatment of endogenous Cushing’s syndrome in adults.
Orphan market exclusivity expiry date: 13 Jan 2030
GB Orphan designation number: PLGB 15266/0029 – 0031/OD1
Jorveza is indicated for the treatment of eosinophilic esophagitis (EoE) in adults (older than 18 years of age).
Orphan market exclusivity expiry date: 10 Jan 2028
GB Orphan designation number: PLGB 08637/0030-0032/OD1
Active substance: ivacaftor, tezacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide (elexacaftor)
Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see section 5.1).
Orphan market exclusivity expiry date: 21 Aug 2030
GB Orphan designation number: PLGB 22352/0012/OD1
GB Orphan Designation number: PLGB 22352/0017/OD1
N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide
Treatment of cystic fibrosis (CF) in patients age six years and older who have a G551D mutation in the CFTR gene
Orphan market exclusivity expiry date: 25 July 2022
GB Orphan designation number: PLGB 22352/0008 – 0011/OD1
N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide
In a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults, adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1)
Orphan market exclusivity expiry date: 25 July 2022
GB Orphan designation number: PLGB 22352/0008/OD1
GB Orphan Designation number: PLGB 22352/0013/OD1
Recombinant human lysosomal acid lipase
Orphan condition: Lysosomal acid lipase deficiency
Kanuma is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) Deficiency.
Orphan market exclusivity expiry date: 1 Sep 2025
GB Orphan designation number: PLGB 31775/0002/OD1
KIMMTRAK is indicated as monotherapy for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma.
Orphan market exclusivity expiry date: 6 June 2032
GB Orphan designation number: PLGB 36781/0001/OD1
Active substance: selumetinib hydrogen sulfate
Authorised orphan indication: Treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above.
Orphan market exclusivity expiry date: 8 Aug 2031
GB Orphan Designation Number: PLGB 17901/0356 - 0357/OD1
Orphan condition: Cushing’s syndrome
Ketoconazole HRA is indicated for the treatment of endogenous Cushing’s syndrome in adults and adolescents above the age of 12 years.
Orphan market exclusivity expiry date: 21 Nov 2024
GB Orphan designation number: PLGB 51757/0002/OD1
Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
Kymriah is indicated for the treatment of paediatric and young adult patients up to 25 years of age with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post transplant or in second or later relapse.
Orphan market exclusivity expiry date: 27 Aug 2028
GB Orphan designation number: PLGB 00101/1101/OD1
Active substance: tisagenlecleucel Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
Orphan condition: Diffuse large B-cell lymphoma
Kymriah is indicated for the treatment of:
Orphan market exclusivity expiry date: 27 Aug 2028
GB Orphan designation number: PLGB 00101/1101/OD2
Kyprolis in combination with daratumumab and dexamethasone, with lenalidomide and dexamethasone, or with dexamethasone alone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.
Orphan market exclusivity expiry date: 23 Nov 2025
GB Orphan designation number: PLGB 13832/0023 – 0025/OD1
Enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha mannosidosis. See sections 4.4 and 5.1.
Orphan market exclusivity expiry date: 27 Mar 2028
GB Orphan designation number: PLGB 08829/0188/OD1
Orphan condition: Cutaneous T-cell lymphoma
Ledaga is indicated for the topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF type CTCL) in adult patients
Orphan market exclusivity expiry date: 7 Mar 2027 GB Orphan designation number: PLGB 15720/0005/OD1
Active substance: Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene
Libmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:
Orphan market exclusivity expiry date: 18 Dec 2030
GB Orphan designation number: PLGB 49055/0002/OD1
Active substance: lutetium (177Lu) oxodotreotide
Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl
Orphan condition: Gastro-entero-pancreatic neuroendocrine tumours
Lutathera is indicated for the treatment of unresectable or metastatic, progressive, well differentiated (G1 and G2), somatostatin receptor positive gastroenteropancreatic neuroendocrine tumours (GEP NETs) in adults.
Orphan market exclusivity expiry date: 28 Sept 2027
GB Orphan designation number: PLGB 35145/0003/OD1
Adenovirus associated viral vector serotype 2 containing the human RPE65 gene
Orphan condition: Inherited retinal dystrophies (initially named Leber’s congenital amaurosis)
Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Orphan market exclusivity expiry date: 5 Dec 2028
GB Orphan designation number: PLGB 00101/1104/OD1
Adenovirus associated viral vector serotype 2 containing the human RPE65 gene
Orphan condition: Inherited retinal dystrophies (initially named retinitis pigmentosa)
Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Orphan market exclusivity expiry date: 5 Dec 2028
GB Orphan designation number: PLGB 00101/1104/OD2
Orphan condition: Mucopolysaccharidosis type VII (Sly syndrome)
Mepsevil is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).
Orphan market exclusivity expiry date: 27 Aug 2028
GB Orphan designation number: PLGB 474793/0002/OD1
Orphan condition: Relapsed or refractory diffuse large B-cell lymphoma
Minjuvi is indicated in combination with lenalidomide followed by Minjuvi monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).
Orphan market exclusivity expiry date: 07 October 2031
GB Orphan Designation Number: PLGB 42338/0016/OD1
Orphan condition: Familial Partial Lipodystrophy
Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients:
Orphan market exclusivity expiry date: 1 Aug 2028
GB Orphan designation number: PLGB 50688/0008-0010/OD1
Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients:
with acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.
Orphan market exclusivity expiry date: 1 Aug 2028
GB Orphan designation number: PLGB 50688/0008-0010/OD2
Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above
Orphan market exclusivity expiry date: 1 Aug 2028
GB Orphan designation number: PLGB 50688/0008-0010/OD3
Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed congenital generalised LD (Berardinelli-Seip syndrome)
Orphan market exclusivity expiry date: 1 Aug 2028
GB Orphan designation number: PLGB 50688/0008-0010/OD4
Orphan condition: Acute myeloid leukaemia
MYLOTARG is indicated for combination therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of patients age 15 years and above with previously untreated, de novo CD33 positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia (APL).
Orphan market exclusivity expiry date: 23 Apr 2028
GB Orphan designation number: PLGB 00057/1591/OD1
Namuscla is indicated for the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders
Orphan market exclusivity expiry date: 20 Dec 2028
GB Orphan designation number: PLGB 35507/0200/OD1
Natpar is indicated as adjunctive treatment of adult patients with chronic hypoparathyroidism who cannot be adequately controlled with standard therapy alone.
Orphan market exclusivity expiry date: 26 Apr 2027
GB Orphan designation number: PLGB 16189/0067 – 0070/OD1
Orphan condition: Follicular thyroid cancer
Nexavar is indicated for the treatment of patients with progressive, locally advanced or metastatic, differentiated (follicular/Hürthle cell) thyroid carcinoma, refractory to radioactive iodine.
Orphan market exclusivity expiry date: 27 May 2024
GB Orphan designation number: PLGB 00010/0701/OD1
Orphan condition: Papillary thyroid cancer
Nexavar is indicated for the treatment of patients with progressive, locally advanced or metastatic, differentiated (papillary) thyroid carcinoma, refractory to radioactive iodine.
Orphan market exclusivity expiry date: 27 May 2024
GB Orphan designation number: PLGB 00010/0701/OD2
Active substance: Concentrate of proteolytic enzymes enriched in bromelain
Partial deep dermal and full thickness burns
NexoBrid is indicated for removal of eschar in adults with deep partial- and full-thickness thermal burns.
Orphan market exclusivity expiry date: 20 Dec 2022
GB Orphan designation number: PLGB 43948/0002 – 0003/OD1
Ngenla is used for the treatment of growth hormone deficiency.
Orphan market exclusivity expiry date: 25 March 2032
GB Orphan Designation Number: PLGB 00057/1712-1713/OD1
2,2’-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid
NINLARO in combination with lenalidomide and dexamethasone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.
Orphan market exclusivity expiry date: 23 Nov 2026
GB Orphan designation number: PLGB 16189/0109 – 0111/OD1
Obiltoxaximab SFL is indicated in combination with appropriate antibacterial drugs in all age groups for treatment of inhalational anthrax due to Bacillus anthracis (see section 5.1).
Obiltoxaximab SFL is indicated in all age groups for post-exposure prophylaxis of inhalational anthrax when alternative therapies are not appropriate or are not available (see section 5.1).
Orphan market exclusivity expiry date: 19 Nov 2030
GB Orphan designation number: PLGB 54280/0001/OD1
Orphan condition: Primary biliary cirrhosis
Orphan condition: Primary biliary cirrhosis
Ocaliva is indicated for the treatment of primary biliary cholangitis (PCB) (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.
Orphan market exclusivity expiry date: 15 Dec 2026
GB Orphan designation number: PLGB 48025/0002 – 0003/OD1
Treatment of metastatic adenocarcinoma of the pancreas, in combination with 5 fluorouracil (5 FU) and leucovorin (LV), in adult patients who have progressed following gemcitabine based therapy.
Orphan market exclusivity expiry date: 18 Oct 2026
GB Orphan designation number: PLGB 05815/0111/OD1
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA
Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
Orphan market exclusivity expiry date: 29 Aug 2028
GB Orphan designation number: PLGB 50597/0002/OD1
Orphan condition: Pulmonary arterial hypertension
Opsumit, as monotherapy or in combination, is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III.
Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease.
Orphan market exclusivity expiry date: 27 Dec 2023
GB Orphan designation number: PLGB 00242/0663/OD1
Orphan condition: Inborn errors in primary bile acid synthesis
Treatment of inborn errors in primary bile acid synthesis due to 3β-Hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ43-Oxosteroid-5β-reductase deficiency in infants, children and adolescents aged 1 month to 18 years and adults.
Orphan market exclusivity expiry date: 16 Sep 2023
GB Orphan designation number: PLGB 44776/0002 – 0003/OD1
Authorised orphan indication: Treatment of moderate (persistent epithelial defect) or severe (corneal ulcer) neurotrophic keratitis in adults.
Orphan market exclusivity date: 10 Jul 2027
GB Orphan Designation Number: PLGB 32162/0002/OD1
Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Oxlumo is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups.
Orphan market exclusivity expiry date: 23 Nov 2030
GB Orphan designation number: PLGB 43942/0003/OD1
Palynziq is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options.
Orphan market exclusivity date: 08 May 2029
GB Orphan Designation Number: PLGB 45814/0008 – 0010/OD1
Orphan condition: Biliary tract cancer
Authorised orphan indication: The treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.
Orphan market exclusivity expiry date: 7 Apr 2031
GB Orphan designation number: PLGB 42338/0008 - 0010/OD1
Orphan condition: Diffuse large B-cell lymphoma
Polivy in combination with bendamustine and rituximab is indicated for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant.
Orphan market exclusivity expiry date: 20 Jan 2030
GB Orphan designation number: PLGB 00031/0912/OD1
Orphan condition: Cutaneous T-cell lymphoma
POTELIGEO is indicated for the treatment of adult patients with mycosis fungoides (MF) or Sézary syndrome (SS) who have received at least one prior systemic therapy.
Orphan market exclusivity expiry date: 26 Nov 2028
GB Orphan designation number: PLGB 50262/0008/OD1
Active substance: letermovir (S)-{8-fluoro-2-2[4-(3-methoxyphenyl)-1-piperazinyl]-3-[2-methoxy-5-(trifluoromethyl)-phenyl]-3,4-dihydro-4-quinazolinyl} acetic acid
Orphan condition: Cytomegalovirus disease reactivation in patients with impaired cell-mediated immunity
PREVYMIS is indicated for prophylaxis of cytomegalovirus (CMV) reactivation and disease in adult CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT).
Consideration should be given to official guidance on the appropriate use of antiviral agents.
Orphan market exclusivity expiry date: 10 Jan 2028
GB Orphan designation number: PLGB 53095/0046 – 0048/OD1
Active substance: mercaptamine (cysteamine bitartrate)
PROCYSBI is indicated for the treatment of proven nephropathic cystinosis. Cysteamine reduces cystine accumulation in some cells (e.g. leukocytes, muscle and liver cells) of nephropathic cystinosis patients and, when treatment is started early, it delays the development of renal failure.
Orphan market exclusivity expiry date: 10 Sep 2023
GB Orphan designation number: PLGB 08829/0190 – 0191/OD1
Chimeric monoclonal antibody against GD2
Qarziba is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures.
In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, Qarziba should be combined with interleukin-2 (IL-2).
Orphan market exclusivity expiry date: 11 May 2027
GB Orphan designation number: PLGB 44185/0005/OD1
Orphan condition: Gastrointestinal stromal tumours
Authorised orphan indication: QINLOCK is indicated for the treatment of adult patients with advanced gastrointestinal stromal tumour (GIST) who have received prior treatment with three or more kinase inhibitors, including imatinib
Orphan market exclusivity expiry date: 20 December 2031
GB Orphan Designation Number: PLGB 55295/0004/OD1
Orphan condition: Carbamoyl-phosphate synthase-1 deficiency
Treatment of carbamoyl-phosphate synthase-1 deficiency:
RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Orphan market exclusivity expiry date: 1 Dec 2027
GB Orphan designation number: PLGB 53487/0001/OD1
Orphan condition: Ornithine carbamoyltransferase deficiency
Treatment of ornithine carbamoyltransferase deficiency:
RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Orphan market exclusivity expiry date: 1 Dec 2027
GB Orphan designation number: PLGB 53487/0001/OD2
RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Orphan market exclusivity expiry date: 1 Dec 2027
GB Orphan designation number: PLGB 53487/0001/OD3
RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Orphan market exclusivity expiry date: 1 Dec 2027
GB Orphan designation number: PLGB 53487/0001/OD4
RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Orphan market exclusivity expiry date: 1 Dec 2027
GB Orphan designation number: PLGB 53487/0001/OD5
Orphan condition: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome)
Treatment of ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome): RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Orphan market exclusivity expiry date: 1 Dec 2027
GB Orphan designation number: PLGB 53487/0001/OD6
Orphan condition: Leber’s hereditary optic neuropathy
Raxone is indicated for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).
Orphan market exclusivity expiry date: 10 Sept 2025
GB Orphan designation number: PLGB 25835/0002/OD1
Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain
Orphan condition: Beta-thalassaemia intermedia and major
Authorised orphan indication: treatment of: - adult patients with transfusion-dependent anaemia associated with Beta-thalassaemia.
Orphan market exclusivity expiry date: 26 Jun 2030
GB Orphan designation number: PLGB 50412/0020 – 0021/OD1
Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain
Treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy
Orphan market exclusivity expiry date: 26 Jun 2030
GB Orphan designation number: PLGB 50412/0020 – 0021/OD2
[gly2] Recombinant human glucagon-like peptide
Orphan condition: Short Bowel Syndrome
Revestive is indicated for the treatment of patients aged 1 year and above with Short Bowel Syndrome. Patients should be stable following a period of intestinal adaptation after surgery.
Orphan market exclusivity expiry date: 4 Sep 2022
GB Orphan designation number: PLGB 16189/0077 – 0078/OD1
Orphan condition: Acute myeloid leukaemia
Rydapt is indicated in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation positive
Orphan market exclusivity expiry date: 20 Sep 2027
GB Orphan designation number: PLGB 00101/1130/OD1
Rydapt is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM AHN), or mast cell leukaemia (MCL).
Orphan market exclusivity expiry date: 20 Sep 2027
GB Orphan designation number: PLGB 00101/1130/OD2
[Nle4, D-Phe7]-alpha-melanocyte stimulating hormone
Scenesse is indicated for prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP).
Orphan market exclusivity expiry date: 29 Dec 2024
GB Orphan designation number: PLGB 30619/0002/OD1
Orphan condition: Chronic myeloid leukaemia
Scemblix is indicated for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase, previously treated with two or more tyrosine kinase inhibitors, and without a known T315I mutation
Orphan market exclusivity expiry date: 15 June 2032
GB Orphan designation number: PLGB 00101/1207/OD1
Treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative and who are inadequately controlled on treatment with another somatostatin analogue.
The 60 mg strength is only to be used in the treatment of acromegaly.
Orphan market exclusivity expiry date: 21 Nov 2024
GB Orphan designation number: PLGB 15266/0032 – 0039/OD1
Orphan condition: Cushing’s disease
Treatment of adult patients with Cushing’s disease for whom surgery is not an option or for whom surgery has failed.
Orphan market exclusivity expiry date: 27 Apr 2022
GB Orphan designation number: PLGB 15266/0032 – 0039/OD2
(1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol
SIRTURO is indicated for use as part of an appropriate combination regimen for pulmonary multidrug resistant tuberculosis (MDR TB) in adults and adolescent patients (12 years to less than 18 years of age and weighing at least 30 kg) when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability (see sections 4.2, 4.4 and 5.1 of the SmPC).
Consideration should be given to official guidance on the appropriate use of antibacterial agents.
Orphan market exclusivity expiry date: 7 Mar 2024
GB Orphan designation number: PLGB 00242/0705/OD1
Orphan condition: Growth hormone deficiency
Authorised orphan indication: Sogroya is indicated for the replacement of endogenous growth hormone in adults with growth hormone deficiency
Orphan market exclusivity expiry date: 21 October 2031
GB Orphan Designation Number: PLGB 04668/0434/OD1
Orphan condition: Atypical haemolytic uremic syndrome
Soliris is indicated in adults and children for the treatment of atypical haemolytic uremic syndrome (aHUS).
Orphan market exclusivity expiry date: 29 Nov 2023
GB Orphan designation number: PLGB 31775/0003/OD1
Soliris is indicated in adults for the treatment of refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive.
Orphan market exclusivity expiry date: 17 Aug 2027
GB Orphan designation number: PLGB 31775/0003/OD2
Orphan condition: Neuromyelitis optica spectrum disorders
Soliris is indicated in adults for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease.
Orphan market exclusivity expiry date: 28 Aug 2029
GB Orphan designation number: PLGB 31775/0003/OD3
Orphan condition: Gastro-entero-pancreatic neuroendocrine tumours
This medicinal product is for diagnostic use only.
After radiolabelling with gallium (68Ga) chloride solution, the solution of gallium (68Ga) edotreotide obtained is indicated for Positron Emission Tomography (PET) imaging of somatostatin receptor overexpression in adult patients with confirmed or suspected well-differentiated gastro-enteropancreatic neuroendocrine tumours (GEP-NET) for localizing primary tumours and their metastases.
Orphan market exclusivity expiry date: 12 Dec 2026
GB Orphan designation number: PLGB 35145/0004/OD1
Antisense oligonucleotide targeted to the SMN2 gene
Orphan condition: 5q spinal muscular atrophy
Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.
Orphan market exclusivity expiry date: 1 Jun 2029
GB Orphan designation number: PLGB 22407/0018/OD1
Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein
Strensiq is indicated for long-term enzyme replacement therapy in patients with paediatric-onset hypophosphatasia to treat the bone manifestations of the disease.
Orphan market exclusivity expiry date: 1 Sep 2025
GB Orphan designation number: PLGB 31775/0004 – 0005/OD1
Active substance: autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence
Orphan condition: Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
Orphan market exclusivity expiry date: 30 May 2028
GB Orphan designation number: PLGB 49055/0001/OD1
Orphan condition: Castleman’s disease
Sylvant is indicated for the treatment of adult patients with multicentric Castleman’s disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus 8 (HHV 8) negative.
Orphan market exclusivity expiry date: 27 May 2024
GB Orphan designation number: PLGB 44185/0006 – 0007/OD1
1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide and ivacaftor
Symkevi is indicated in a combination regimen with ivacaftor tablets for the treatment of patients with cystic fibrosis (CF) aged 6 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T.
Orphan market exclusivity expiry date: 6 Nov 2028
GB Orphan designation number: PLGB 22352/0003/OD1
Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein
TAKHZYRO is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older.
Orphan market exclusivity expiry date: 26 Nov 2028
GB Orphan designation number: PLGB 16189/0084/OD1
Active substance: autologous anti-CD19-transduced CD3+ cells
Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured
Orphan condition: Mantle cell lymphoma
Tecartus is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor
Orphan market exclusivity expiry date: 15 Dec 2030
GB Orphan designation number: PLGB 11972/0045/OD1
Phosphorothioate oligonucleotide targeted to transthyretin
Tegsedi is indicated for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
Orphan market exclusivity expiry date: 10 Jul 2028
GB Orphan designation number: PLGB 51704/0002/OD1
Orphan condition: Pseudomonas aeruginosa lung infection in cystic fibrosis
TOBI Podhaler is indicated for the suppressive therapy of chronic pulmonary infection due to Pseudomonas aeruginosa in adults and children aged 6 years and older with cystic fibrosis. Consideration should be given to official guidance on the appropriate use of antibacterial agents.
Orphan market exclusivity expiry date: 25 Jul 2023
GB Orphan designation number: PLGB 46302/0222/OD1
(3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid
Orphan condition: Duchenne muscular dystrophy
Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing
Orphan market exclusivity expiry date: 5 Aug 2024
GB Orphan designation number: PLGB 44221/0002 – 0004/OD1
Orphan condition: Haematopoietic progenitor cell transplantation
Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with malignant and non malignant diseases, and in paediatric patients older than one month with malignant diseases.
Orphan market exclusivity expiry date: 24 June 2029
GB Orphan designation number: PLGB 11587/0118 - 0119/OD1
Orphan condition: Chronic thromboembolic pulmonary hypertension
Trepulmix is indicated for the treatment of adult patients with WHO Functional Class (FC) III or IV and:
Orphan market exclusivity expiry date: 7 Apr 2030
GB Orphan designation number: PLGB 51174/0001 – 0004/OD1
Treatment of severe vernal keratoconjunctivitis (VKC) in children from 4 years of age and adolescents
Orphan market exclusivity expiry date: 10 Jul 2030
GB Orphan designation number: PLGB 16508/0028/OD1
Orphan condition: Mucopolysaccharidosis, type IVA (Morquio A Syndrome)
Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.
Orphan market exclusivity expiry date: 30 Apr 2024
GB Orphan designation number: PLGB 45814/0007/OD1
Renal angiomyolipoma associated with tuberous sclerosis complex (TSC) Votubia is indicated for the treatment of adult patients with renal angiomyolipoma associated with TSC who are at risk of complications (based on factors such as tumour size or presence of aneurysm, or presence of multiple or bilateral tumours) but who do not require immediate surgery.
The evidence is based on analysis of change in sum of angiomyolipoma volume.
Subependymal giant cell astrocytoma (SEGA) associated with TSC Votubia is indicated for the treatment of adult and paediatric patients with SEGA associated with TSC who require therapeutic intervention but are not amenable to surgery.
The evidence is based on analysis of change in SEGA volume. Further clinical benefit, such as improvement in disease related symptoms, has not been demonstrated.
Refractory seizures associated with tuberous sclerosis complex (TSC) Votubia is indicated as adjunctive treatment of patients aged 2 years and older whose refractory partial onset seizures, with or without secondary generalisation, are associated with TSC.
Orphan market exclusivity expiry date: 6 Sep 2023
GB Orphan designation number: PLGB 00101/1162 – 1168/OD1
VPRIV is indicated for long-term enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease.
Orphan market exclusivity expiry date: 30 Aug 2022
GB Orphan designation number: PLGB 16189/0088/OD1
N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate
Orphan condition: ATTR - amyloidosis
Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment.
Vyndaqel is indicated for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
Orphan market exclusivity expiry date: 19 Feb 2030
GB Orphan designation number: PLGB 00057/1660/OD2
Liposomal combination of cytarabine and daunorubicin
Orphan condition: Acute myeloid leukaemia
Vyxeos is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).
Orphan market exclusivity expiry date: 27 Aug 2028
GB Orphan designation number: PLGB 31626/0004/OD1
1-{3-[3-(4-chlorophenyl)propoxy]propyl}piperidine, hydrochloride
Wakix is indicated in adults for the treatment of narcolepsy with or without cataplexy.
Orphan market exclusivity expiry date: 4 Apr 2026
GB Orphan designation number: PLGB 26351/0005 – 0006/OD1
Phosphorothioate oligonucleotide targeted to apolipoprotein C-III
Orphan condition: Familial chylomicronemia syndrome
Waylivra is indicated as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate
Orphan market exclusivity expiry date: 8 May 2029
GB Orphan designation number: PLGB 51704/0003/OD1
(S)-ethyl 2-amino-3-(4-(2-amino-6-((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate
Xermelo is indicated for the treatment of carcinoid syndrome diarrhoea in combination with somatostatin analogue (SSA) therapy in adults inadequately controlled by SSA therapy.
Orphan market exclusivity expiry date: 20 Sep 2027
GB Orphan designation number: PLGB 28247/0008/OD1
Orphan condition: Acute myeloid leukaemia
Xospata is indicated as monotherapy for the treatment of adult patients who have relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation.
Orphan market exclusivity expiry date: 28 Oct 2029
GB Orphan designation number: PLGB 00166/0425/OD1
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor
Orphan condition: Diffuse large B cell lymphoma
YESCARTA is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy
Orphan market exclusivity expiry date: 27 Aug 2028
GB Orphan designation number: PLGB 11972/0044/OD1
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor
Orphan condition: Primary mediastinal large B-cell lymphoma
YESCARTA is indicated for the treatment of adult patients with relapsed or refractory primary mediastinal large B-cell lymphoma (PMBCL) after two or more lines of systemic therapy.
Orphan market exclusivity expiry date: 27 Aug 2028
GB Orphan designation number: PLGB 11972/0044/OD2
(3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt
Orphan market exclusivity expiry date: 20 Nov 2027
GB Orphan designation number: PLGB 19494/0294/OD1
Adeno-associated viral vector serotype 9 containing the human SMN gene
Orphan condition: Spinal muscular atrophy
Zolgensma is indicated for the treatment of:
Orphan market exclusivity expiry date: 18 May 2030
GB Orphan designation number: PLGB 53139/0002/OD1
Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
Orphan condition: Beta-thalassaemia intermedia and major
Zynteglo is indicated for the treatment of patients 12 years and older with transfusion dependent β thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA) matched related HSC donor is not available
Orphan market exclusivity expiry date: 4 Jun 2029
GB Orphan designation number: PLGB 51370/0001/OD1
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