Question

ABECMA

Active substance: idecabtagene vicleucel    

Orphan condition: Multiple myeloma

Authorised orphan indication: Abecma is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy

Orphan market exclusivity expiry date: 23 June 2032

GB Orphan Designation Number: PLGB 50412/0023/OD1

ADAKVEO

Active substance: crizanlizumab

Orphan condition: Sickle cell disease

Authorised orphan indication:

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 October 2030

GB Orphan Designation Number: PLGB 00101/1191/OD1

Comments

Oxlumo
Active substance: lumasiran

Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues

Orphan condition: Primary hyperoxaluria

Authorised orphan indication:

Oxlumo is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups.

Orphan market exclusivity expiry date: 23 November 2030

GB Orphan designation number: PLGB 43942/0003/OD1

Palynziq
Active substance: pegvaliase

Orphan condition: hyperphenylalaninaemia

Authorised orphan indication:

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: 8 May 2029

GB Orphan Designation Number: PLGB 45814/0008 – 0010/OD1

Pemazyre
Active substance: pemigatinib

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 April 2031

GB Orphan designation number: PLGB 42338/0008 - 0010/OD1

Pombiliti 105 mg powder for concentrate for solution for infusion
Active substance: CIPAGLUCOSIDASE ALFA

Orphan condition: Glycogen storage disease type II (Pompe’s disease)

Authorised orphan indication: Long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency

Orphan market exclusivity expiry date: 30 June 2033

GB Orphan designation number: PLGB 25823/0003/OD1

Polivy
Active substance: polatuzumab vedotin

Orphan condition: Diffuse large B-cell lymphoma

Authorised orphan indication:

Polivy in combination with rituximab, cyclophosphamide, doxorubicin, and prednisone (R-CHP) is indicated for the treatment of adult patients with previously untreated diffuse large B-cell lymphoma (DLBCL).

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 January 2030

GB Orphan designation number: PLGB 00031/0912/OD1

Poteligeo
Active substance: mogamulizumab

Orphan condition: Cutaneous T-cell lymphoma

Authorised orphan indication:

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 November 2028

GB Orphan designation number: PLGB 50262/0008/OD1

PREVYMIS
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

Authorised orphan indication:

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 January 2028

GB Orphan designation number: PLGB 53095/0046 – 0048/OD1

PYRUKYND
Active substance: Mitapivat sulfate

Potent, allosteric activator of wild-type red blood cell (RBC)-specific form of pyruvate kinase (PKR) and a range of mutant PKR enzymes. Mitapivat targets the underlying enzymatic defect that causes haemolysis in pyruvate kinase deficiency by restoring the activity of mutant forms of PKR.

Orphan condition: pyruvate kinase deficiency (PK deficiency)

Authorised orphan indication:

Pyrukynd is indicated for the treatment of pyruvate kinase deficiency (PK deficiency) in adult patients

Orphan market exclusivity expiry date: 29 November 2032

GB Orphan designation number: PLGB 52779/0001-0005OD1

Qarziba
Active substance: dinutuximab beta

Chimeric monoclonal antibody against GD2

Orphan condition: Neuroblastoma

Authorised orphan indication:

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

QINLOCK
Active substance: ripretinib

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

Ravicti
Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Carbamoyl-phosphate synthase-1 deficiency

Authorised orphan indication:

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 December 2027

GB Orphan designation number: PLGB 53487/0001/OD1

Ravicti
Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Ornithine carbamoyltransferase deficiency

Authorised orphan indication:

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 December 2027

GB Orphan designation number: PLGB 53487/0001/OD2

Ravicti
Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Citrullinaemia type 1

Authorised orphan indication:

Treatment of citrullinaemia type 1:

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 December 2027

GB Orphan designation number: PLGB 53487/0001/OD3

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Ravicti
Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Argininosuccinic aciduria

Authorised orphan indication:

Treatment of argininosuccinic aciduria:

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 December 2027

GB Orphan designation number: PLGB 53487/0001/OD4

Ravicti
Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Hyperargininaemia

Authorised orphan indication:

Treatment of hyperargininaemia:

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 December 2027

GB Orphan designation number: PLGB 53487/0001/OD5

Ravicti
Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome)

Authorised orphan indication:

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 December 2027

GB Orphan designation number: PLGB 53487/0001/OD6

Raxone
Active substance: idebenone

Orphan condition: Leber’s hereditary optic neuropathy

Authorised orphan indication:

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 September 2025

GB Orphan designation number: PLGB 08829/0204/OD1

Reblozyl
Active substance: luspatercept

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 June 2030

GB Orphan designation number: PLGB 50412/0020 – 0021/OD1

Reblozyl
Active substance: luspatercept

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: Myelodysplastic syndromes

Authorised orphan indication:

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 June 2030

GB Orphan designation number: PLGB 50412/0020 – 0021/OD2

REZUROCK
Active substance: belumosudil mesilate

Orphan condition: Graft versus host disease (GVHD)

Authorised orphan indication:

Rezurock is indicated for the treatment of patients aged 12 years and older with chronic graft-versus-host disease (chronic GVHD) who have received at least two prior lines of systemic therapy.

Orphan market exclusivity date:  6 July 2032

GB Orphan Designation Number: PLGB 53904/0001/OD1

Rezzayo
Active substance: REZAFUNGIN ACETATE  

Orphan condition: Treatment of invasive candidiasis

Authorised orphan indication:  Treatment of invasive candidiasis in adult

Orphan market exclusivity expiry date: 29/01/2034

GB Orphan Designation Number: PLGB 16950/0390/OD1

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Rydapt
Active substance: midostaurin

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication:

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 September 2027

GB Orphan designation number: PLGB 00101/1130/OD1

Rystiggo 140 mg/ml solution for injection  
Active substance: ROZANOLIXIZUMAB

Orphan condition: Myasthenia gravis

Authorised orphan indication: as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.

Orphan market exclusivity expiry date: 07/03/2034

GB Orphan designation number: PLGB 00039/0808/OD1

Rydapt
Active substance: midostaurin

Orphan condition: Mastocytosis

Authorised orphan indication:

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 September 2027

GB Orphan designation number: PLGB 00101/1130/OD2

SCENESSE
Active substance: afamelanotide

[Nle4, D-Phe7]-alpha-melanocyte stimulating hormone

Orphan condition: Erythropoietic protoporphyria

Authorised orphan indication:

Scenesse is indicated for prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP).

Orphan market exclusivity expiry date: 29 December 2024

GB Orphan designation number: PLGB 30619/0002/OD1

Scemblix
Active substance: Asciminib

Orphan condition: Chronic myeloid leukaemia

Authorised orphan indication:

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

Sogroya
Active substance: somapacitan

Orphan condition: Growth hormone deficiency

Authorised orphan indication:

Sogroya is indicated for the replacement of endogenous growth hormone (GH) in children aged 3 years and above, and adolescents with growth failure due to growth hormone deficiency (paediatric GHD), and in adults with growth hormone deficiency (adult GHD).

Orphan market exclusivity expiry date: 21 October 2031

GB Orphan Designation Number: PLGB 04668/0434/OD1

Soliris
Active substance: eculizumab

Orphan condition: Myasthenia gravis

Authorised orphan indication:

Soliris is indicated for the treatment of refractory generalized myasthenia gravis (gMG) in patients aged 6 years and above who are antiacetylcholine receptor (AChR) antibody-positive

Orphan market exclusivity expiry date: 17 August 2027

GB Orphan designation number: PLGB 31775/0003/OD2

Soliris
Active substance: eculizumab

Orphan condition: Neuromyelitis optica spectrum disorders

Authorised orphan indication:

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 August 2029

GB Orphan designation number: PLGB 31775/0003/OD3

SomaKit TOC
Active substance: edotreotide

Gallium (68Ga)-edotreotide

Orphan condition: Gastro-entero-pancreatic neuroendocrine tumours

Authorised orphan indication:

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 December 2026

GB Orphan designation number: PLGB 35145/0004/OD1

Spexotras
Active substance: Trametinib

Orphan condition: Glioma

Authorised orphan indication:

Low-grade glioma

Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.

High-grade glioma

Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment.

Orphan market exclusivity expiry date: 13 February 2036

GB Orphan designation number: PLGB 00101/1227/OD1

Spinraza
Active substance: nusinersen

Antisense oligonucleotide targeted to the SMN2 gene

Orphan condition: 5q spinal muscular atrophy

Authorised orphan indication:

Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.

Orphan market exclusivity expiry date: 1 June 2029

GB Orphan designation number: PLGB 22407/0018/OD1

Strensiq
Active substance: asfotase alfa

Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein

Orphan condition: Hypophosphatasia

Authorised orphan indication:

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 September 2025

GB Orphan designation number: PLGB 31775/0004 – 0005/OD1

Strimvelis
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

Authorised orphan indication:

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

Symkevi
Active substance: tezacaftor/ivacaftor

Orphan condition: Cystic fibrosis

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

Authorised orphan indication:

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 November 2028

GB Orphan designation number: PLGB 22352/0003/OD1

TAKHZYRO
Active substance: lanadelumab

Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein

Orphan condition: Hereditary angioedema

Authorised orphan indication:

TAKHZYRO is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 2 years and older.

Orphan market exclusivity expiry date: 26 November 2030

GB Orphan designation number: PLGB 54937/0017/OD1

TAVNEOS
Active substance: tavacopan

Orphan condition: Granulomatosis with polyangiitis

Authorised orphan indication: Tavneos, In combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA)

Orphan market exclusivity expiry date: 5 May 2032

GB Orphan Designation Number: PLGB 50784/0008/OD1

TAVNEOS
Active substance: tavacopan

Orphan condition: Microscopic polyangiitis

Authorised orphan indication: Tavneos, In combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA)

Orphan market exclusivity expiry date: 5 May 2032

GB Orphan Designation Number: PLGB 50784/0008/OD2

Tecartus
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: Acute lymphoblastic leukaemia

Authorised orphan indication:

Tecartus is indicated for the treatment of adult patients 26 years of age and above with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL).

Orphan market exclusivity expiry date: 7 November 2032

GB Orphan designation number: PLGB 11972/0045/OD2

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Tecartus
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

Authorised orphan indication:

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: 14 December 2030

GB Orphan designation number: PLGB 11972/0045/OD1

Tegsedi
Active substance: inotersen

Phosphorothioate oligonucleotide targeted to transthyretin

Orphan condition: ATTR amyloidosis

Authorised orphan indication:

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 July 2028

GB Orphan designation number: PLGB 51704/0002/OD1

TIBSOVO
Active substance: ivosidenib

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication: In combination with azacitidine for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy (see section 5.1)

Orphan market exclusivity expiry date: 5 July 2033

GB Orphan Designation Number: PLGB 05815/0120/OD1

TIBSOVO
Active substance:  ivosidenib

Orphan condition: Biliary tract cancer

Authorised orphan indication: as monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy (see section 5.1).

Orphan market exclusivity expiry date: 5 July 2033

GB Orphan Designation Number: PLGB 05815/0120/OD2

Trecondi
Active substance: treosulfan

Orphan condition: Haematopoietic progenitor cell transplantation

Authorised orphan indication:

Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients and in paediatric patients older than one month with malignant and non malignant diseases.

Orphan market exclusivity expiry date: 24 June 2031

GB Orphan designation number: PLGB 11587/0118 - 0119/OD1

Trepulmix
Active substance: treprostinil sodium

Orphan condition: Chronic thromboembolic pulmonary hypertension

Authorised orphan indication:

Trepulmix is indicated for the treatment of adult patients with WHO Functional Class (FC) III or IV and:

inoperable chronic thromboembolic pulmonary hypertension (CTEPH), or
persistent or recurrent CTEPH after surgical treatment to improve exercise capacity.
Orphan market exclusivity expiry date: 7 April 2030

GB Orphan designation number: PLGB 51174/0001 – 0004/OD1

UPSTAZA
Active substance: eladocagene exuparvovec

Orphan condition: Aromatic L-amino acid decarboxylase deficiency

Authorised orphan indication:

For the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype.

Orphan market exclusivity expiry date: 17 November 2034

GB Orphan designation number: PLGB 44221/0006/OD1V

Vanflyta
Active substance: Quizartinib

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication: In combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by VANFLYTA single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3-ITD positive

Orphan market exclusivity expiry date: 11/03/2034

GB Orphan Designation Number: PLGB 08265/0047-0048/OD1

Verkazia
Active substance: ciclosporin

Orphan condition: Vernal keratoconjunctivitis

Authorised orphan indication:

Treatment of severe vernal keratoconjunctivitis (VKC) in children from 4 years of age and adolescents

Orphan market exclusivity expiry date: 10 July 2030

GB Orphan designation number: PLGB 16508/0028/OD1

Voraxaze 1000 units powder for solution for injection
Active substance: GLUCARPIDASE

Orphan condition: Methotrexate-induced nephropathy

Authorised orphan indication: Voraxaze is indicated to reduce toxic plasma methotrexate concentration in adults and children (aged 28 days and older) with delayed methotrexate elimination.

Orphan market exclusivity expiry date: 15 June 2035

GB Orphan designation number: PLGB 18442/0002/OD1

Vyloy
Active substance: zolbetuximab

Orphan condition: malignant neoplasm of stomach

Authorised orphan indication: Vyloy, in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 -negative gastric or gastro-oesophageal junction adenocarcinoma whose tumours are Claudin 18.2 positive

Orphan market exclusivity expiry date: 14/08/2034

GB Orphan Designation Number: PLGB 00166/0439/OD1

Vyndaqel
Active substance: tafamidis

N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate

Orphan condition: Senile systemic amyloidosis

Authorised orphan indication:

treatment of wild-type transthyretin amyloidosis in adult patients with cardiomyopathy

Orphan market exclusivity expiry date: 19 February 2030

GB Orphan designation number: PLGB 00057/1660/OD2

Note: Wild-type transthyretin amyloid is also known as senile systemic amyloidosis and Hereditary transthyretin amyloid is also known as familial amyloidosis. The protection for hereditary amyloid ended on 18 Nov 2021.

Vyvgart 20 mg/ml Concentrate for solution for infusion
Active substance: EFGARTIGIMOD ALFA

Orphan condition: Myasthenia Gravis (gMG)

Authorised orphan indication:

Treatment of adult patients with generalised Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

Orphan market exclusivity expiry date: 14 March 2033

GB Orphan designation number: PLGB 47104/0004/OD1Vyvgart 20 mg/ml Concentrate for solution for infusion

Active substance: EFGARTIGIMOD ALFA

Orphan condition: Myasthenia Gravis (gMG)

Authorised orphan indication:

Treatment of adult patients with generalised Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

Orphan market exclusivity expiry date: 14 March 2033

GB Orphan designation number: PLGB 47104/0004/OD1

Vyxeos liposomal
Active substance: daunorubicin / cytarabine

Liposomal combination of cytarabine and daunorubicin

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication:

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 August 2028

GB Orphan designation number: PLGB 31626/0004/OD1

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Wainzua
Wainzua 45 mg solution for injection in pre-filled pen

Active substance: EPLONTERSEN

Orphan condition: Transthyretin-mediated amyloidosis

Authorised orphan indication: Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv amyloidosis) in adult patients with Stage 1 and 2 polyneuropathy.

Orphan market exclusivity expiry date: 14 October 2034

GB Orphan designation number: PLGB 17901/0377/OD1

Wakix
Active substance: pitolisant hydrochloride

1-{3-[3-(4-chlorophenyl)propoxy]propyl}piperidine, hydrochloride

Orphan condition: Narcolepsy

Authorised orphan indication:

Wakix is indicated in adults for the treatment of narcolepsy with or without cataplexy.

Orphan market exclusivity expiry date: 4 April 2026

GB Orphan designation number: PLGB 26351/0005 – 0006/OD1

Welireg
Active substance: BELZUTIFAN

Orphan condition: von Hippel-Lindau (VHL) disease

Authorised orphan indication:

Treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable or undesirable.

Orphan market exclusivity expiry date: 30 May 2032

GB Orphan designation number: PLGB 53095/0087/OD1

XENPOZYME
Active substance: Olipudase alfa

Orphan condition: Acid sphingomyelinase deficiency

Authorised orphan indication:

Enzyme replacement therapy for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency in paediatric and adult patients with type A/B or type B.

Orphan market exclusivity expiry date: 31 July 2032

GB Orphan designation number: PLGB 04425/0901/OD1

Waylivra
Active substance: volanesorsen

Phosphorothioate oligonucleotide targeted to apolipoprotein C-III

Orphan condition: Familial chylomicronemia syndrome

Authorised orphan indication:

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

Xermelo
Active substance: telotristat

(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

Orphan condition: Carcinoid syndrome

Authorised orphan indication:

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 September 2027

GB Orphan designation number: PLGB 28247/0008/OD1

Xospata
Active substance: gilteritinib

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication:

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 October 2029

GB Orphan designation number: PLGB 00166/0425/OD1

Yescarta
Active substance: axicabtagene ciloleucel

Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor

Orphan condition: Diffuse large B cell lymphoma

Authorised orphan indication:

treatment of adult patients with diffuse large B cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL) that relapses within 12 months from completion of, or is refractory to, first-line chemoimmunotherapy.

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 August 2028

GB Orphan designation number: PLGB 11972/0044/OD1

Yescarta
Active substance: axicabtagene ciloleucel

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

Authorised orphan indication:

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 August 2028

GB Orphan designation number: PLGB 11972/0044/OD2

Yescarta
Active substance: axicabtagene ciloleucel

Orphan condition: Follicular lymphoma

Authorised orphan indication: treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy’

Orphan market exclusivity expiry date: 9 August 2032

GB Orphan Designation Number: PLGB 11972/0044/OD3

Yorvipath 168 micrograms/0.56 mL solution for injection in pre filled pen
Active substance: PALOPEGTERIPARATIDE

Orphan condition: Hypoparathyroidism

Authorised orphan indication: Treatment of hypoparathyroidism.

Orphan market exclusivity expiry date: 23/04/2034

GB Orphan designation number: PLGB 51127/0001-0003/OD1

Yorvipath 294 micrograms/0.98 mL solution for injection in pre filled pen
Active substance: PALOPEGTERIPARATIDE

Orphan condition: Hypoparathyroidism

Authorised orphan indication: Treatment of hypoparathyroidism.

Orphan market exclusivity expiry date: 23/04/2034

GB Orphan designation number: PLGB 51127/0001-0003/OD1

Yorvipath 420 micrograms/1.4 mL solution for injection in pre filled pen
Active substance: PALOPEGTERIPARATIDE

Orphan condition: Hypoparathyroidism

Authorised orphan indication: Treatment of hypoparathyroidism.

Orphan market exclusivity expiry date: 23/04/2034

GB Orphan designation number: PLGB 51127/0001-0003/OD1

Zejula
Active substance: niraparib

(3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt

Orphan condition: Ovarian cancer

Authorised orphan indication:

Zejula is indicated:

as monotherapy for the maintenance treatment of adult patients with advanced epithelial (FIGO Stages III and IV) high-grade ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy.
as monotherapy for the maintenance treatment of adult patients with platinum sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum based chemotherapy
Orphan market exclusivity expiry date: 20 November 2027

GB Orphan designation number: PLGB 19494/0294/OD1

Zokinvy
Active substance: lonafarnib

Orphan condition: Hutchinson-Gilford progeria syndrome

Authorised orphan indication:

Treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation.

Orphan market exclusivity expiry date: 24 August 2034

GB Orphan designation number: PLGB 56709/0001-0002/ OD1

Zolgensma
Active substance: onasemnogene abeparvovec

Adeno-associated viral vector serotype 9 containing the human SMN gene

Orphan condition:

Spinal muscular atrophy

Authorised orphan indication:

Zolgensma is indicated for the treatment of:

patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or
patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
Orphan market exclusivity expiry date: 18 May 2030

GB Orphan designation number: PLGB 53139/0002/OD1

Ztalmy 50 mg/ml Oral suspension  
Active substance: GANAXOLONE  

Orphan condition: Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder

Authorised orphan indication: indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older.

Orphan market exclusivity expiry date: 07/03/2034

GB Orphan designation number: PLGB 55682/0001/OD1

Zynteglo
Active substance: betibeglogene autotemcel

Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene

Orphan condition: Beta-thalassaemia intermedia and major

Authorised orphan indication:

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 June 2029

GB Orphan designation number: PLGB 51370/0001/OD1