Orphan Registration-GOV.UK |

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The publication is available at https://www.gov.uk/government/publications/orphan-registered-medicinal-products/orphan-register

Orphan registered products are listed in alphabetical order of product names.

You can search the list by pressing "CTRL F" and entering a keyword or phrase.

Where you are instructed to check the numbering section for more information, you can do this in the Summary of Product Characteristics (SmPC) for each product.

Orphan disease: sickle cell disease

Adakveo is indicated for the prevention of recurrent vascular occlusive crisis (VOC) in patients 16 years of age and older with sickle cell disease. It can be used as an add-on therapy to hydroxyurea/hydroxyurea (HU/HC) or as a monotherapy for patients with unsuitable or insufficient HU/HC.

Orphan drug market exclusive date: October 29, 2030

GB Orphan Drug Number: PLGB 00101/1191/OD1

Anti-human CD30 monoclonal antibody covalently linked to cytotoxin monomethyl auristatin E

Orphan disease: systemic anaplastic large cell lymphoma.

ADCETRIS in combination with cyclophosphamide, doxorubicin and prednisone (CHP) is suitable for adult patients with previously untreated systemic anaplastic large cell lymphoma (sALCL).

ADCETRIS is suitable for the treatment of adult patients with relapsed or refractory sALCL.

The expiration date of the orphan drug market exclusivity: October 30, 2022

GB Orphan Drug Number: PLGB 16189/0093/OD1

Anti-human CD30 monoclonal antibody covalently linked to cytotoxin monomethyl auristatin E

ADCETRIS is indicated for adult patients with previously untreated CD30 stage IV Hodgkin's lymphoma (HL) combined with doxorubicin, vinblastine, and dacarbazine (AVD).

ADCETRIS is indicated for the treatment of adult patients with CD30 HL who are at increased risk of recurrence or progression after autologous stem cell transplantation (ASCT).

ADCETRIS is suitable for the treatment of adult patients with relapsed or refractory CD30 Hodgkin lymphoma (HL):

The expiration date of the orphan drug market exclusivity: October 30, 2022

GB Orphan Drug Number: PLGB 16189/0093/OD2

Anti-human CD30 monoclonal antibody covalently linked to cytotoxin monomethyl auristatin E

Orphan disease: skin T-cell lymphoma

ADCETRIS is suitable for the treatment of adult patients with CD30 cutaneous T-cell lymphoma (CTCL) after at least one previous systemic treatment

Orphan drug market exclusivity expiration date: December 19, 2027

GB Orphan Drug Number: PLGB 16189/0093/OD3

4,6-Diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridin-3-yl]-5-pyrimidinyl(methyl)carbamate ester

Orphan disease: chronic thromboembolic pulmonary hypertension (CTEPH)

Adempas is suitable for the treatment of adult patients with WHO functional class (FC) II to III

Adempas is used as a monotherapy or in combination with endothelin receptor antagonists and is suitable for the treatment of adult patients with pulmonary hypertension (PAH) of WHO functional class (FC) II to III to improve exercise capacity.

Efficacy has been shown in the PAH population, including the etiology of idiopathic or hereditary PAHs or PAHs associated with connective tissue diseases.

The expiration date of the orphan drug market exclusivity: March 31, 2024

GB Orphan Drug Number: PLGB 00010/0670 – 0674/OD1

Active substance: expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue

Alofisel is suitable for the treatment of complex perianal fistulas in adult patients with inactive/mildly active luminal Crohn’s disease, when the fistula does not respond adequately to at least one conventional or biological therapy. Alofisel should be used after fistula conditioning.

Orphan drug market exclusivity expiration date: March 27, 2028

GB Orphan Drug Number: PLGB 16189/0094/OD1

Recombinant fusion protein consisting of human coagulation factor IX linked to the Fc domain of human IgG1

Orphan disease: hemophilia B (congenital factor IX deficiency)

Treatment and prevention of bleeding in patients with hemophilia B (congenital factor IX deficiency).

ALPROLIX can be used in all age groups.

Orphan drug market exclusivity expiration date: May 13, 2026

GB Orphan Drug Number: PLGB 30941/0003 – 0007/OD1

AMGLIDIA is suitable for the treatment of neonatal diabetes, suitable for newborns, infants and children.

Sulfonylureas such as AMGLIDIA have been shown to be effective in patients with mutations in genes encoding β-cell ATP-sensitive potassium channels and transient neonatal diabetes related to chromosome 6q24.

Orphan drug market exclusivity expiration date: May 28, 2028

GB Orphan Drug Number: PLGB 50687/0001 – 0004/OD1

Orphan disease: non-tuberculous mycobacterial lung disease

ARIKAYCE liposomes are suitable for the treatment of nontuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium complex (MAC). Adults with limited treatment options but no cystic fibrosis.

The expiration date of the orphan drug market exclusivity: October 28, 2030

Orphan Drug 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 disease: gastrointestinal stromal tumor

AYVAKYT is designated as a monotherapy for the treatment of unresectable or metastatic gastrointestinal stromal tumor (GIST) adult patients with platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation

The expiration date of the orphan drug market exclusivity: September 25, 2030

Orphan Number: PLGB 52115/0001 – 0003/0D1

Orphan disease: B-cell acute lymphoblastic leukemia

BESPONSA is designated as a monotherapy for the treatment of relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL) in adults. Adult patients with Philadelphia chromosome-positive (Ph) relapsed or refractory B-cell precursor ALL should be treated with at least one tyrosine kinase inhibitor (TKI) that has failed.

Orphan drug market exclusivity expiration date: July 3, 2027

GB Orphan Drug Number: PLGB 00057/1546/OD1

Humanized monoclonal antibody targeting B cell maturation antigen conjugated with maleimidohexanoyl monomethyl auristatin F

BLENREP is designated as a monotherapy for the treatment of multiple myeloma in adults. These patients have received at least four treatments in the past, and their disease is related to at least one proteasome inhibitor, one immunomodulator and one anti-CD38 monoclonal antibody Have drug resistance and suffer from disease progression that was proven in the last treatment

The expiration date of the orphan drug market exclusivity: August 26, 2030

Orphan Drug Number: PLGB 19494/0296/OD1

Orphan disease: acute lymphoblastic leukemia

BLINCYTO is designated as a monotherapy for the treatment of Philadelphia chromosome-negative CD19-positive relapsed or refractory B precursor acute lymphoblastic leukemia (ALL) adults.

BLINCYTO is designated as a monotherapy for the treatment of Philadelphia chromosome-negative CD19-positive B precursor ALL in the first or second complete remission of adults with minimal residual disease (MRD) greater than or equal to 0.1%.

BLINCYTO is indicated for monotherapy in the treatment of pediatric patients with Philadelphia chromosome-negative CD19-positive B-precursor ALL aged 1 year or older who are refractory or relapsed after receiving at least two previous treatments or relapsed after receiving previous allogeneic hematopoietic stem cell transplantation.

Orphan drug market exclusivity expiration date: November 25, 2025

GB Orphan Drug Number: PLGB 13832/0018/OD1

Orphan disease: neuronal ceroid lipofuscinosis type 2

Brineura is used to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.

The expiration date of the orphan drug market exclusivity period: June 1, 2027

GB Orphan Drug Number: PLGB 45814/0001/OD1

Bronchitol is indicated for the treatment of cystic fibrosis (CF) in adults 18 years of age and older as an add-on therapy to the best standard of care.

The expiration date of the orphan drug market exclusivity: April 18, 2022

GB Orphan Drug Number: PLGB 27944/0002/OD1

Orphan disease: progressive familial intrahepatic cholestasis

Approved Orphan Drug Indications: Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients 6 months or more.

Orphan drug market exclusivity expiration date: August 4, 2031

GB Orphan Drug Number: PLGB 36216/0001-0004/OD1

Nanobody against human von Willebrand factor A1 domain

Orphan disease: thrombotic thrombocytopenic purpura

Cablivi is suitable for the treatment of adults and adolescents 12 years of age and older weighing at least 40 kg with acquired thrombotic thrombocytopenic purpura (aTTP), combined with plasma exchange and immunosuppression.

The expiration date of the orphan drug market exclusivity: September 4, 2030

GB Orphan Drug 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-tartrate

Cerdelga is suitable for long-term treatment of adult patients with type 1 Gaucher disease (GD1), these patients are CYP2D6 poor metabolizers (PMs), intermediate metabolizers (IMs) or strong metabolizers (EMs).

Orphan drug market exclusivity expiration date: January 21, 2025

GB Orphan Drug Number: PLGB 04425/0763/OD1

Orphan disease: a congenital error in the synthesis of primary bile acids

Chenodeoxycholic acid is suitable for the treatment of primary bile acid synthesis caused by sterol 27 hydroxylase deficiency (manifested as cerebral tendon xanthomatosis (CTX)) in infants, children and adolescents and adults from 1 month to 18 years old Congenital error

Orphan drug market exclusivity expiration date: April 12, 2027

GB Orphan Drug Number: PLGB 44975/0001/OD1

Active substance: human coagulation factor X

Orphan status: hereditary factor X deficiency

Coagadex is suitable for the treatment and prevention of bleeding events and the perioperative management of patients with hereditary factor X deficiency.

Coagadex is suitable for all age groups.

The expiration date of the orphan drug market exclusivity period: March 18, 2028

GB Orphan Drug 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

Orphan disease: medullary thyroid carcinoma

COMETRIQ is suitable for the treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid cancer.

For patients whose RET mutation status is unknown or negative during transfection, the possible lower benefit should be considered before making individual treatment decisions.

The expiration date of the orphan drug market exclusivity: March 26, 2024

GB Orphan Drug Number: PLGB 28247/0004 – 0005/OD1

Treatment of mucormycosis in patients who are not suitable for amphotericin B

The official guidelines for the proper use of antifungal agents should be considered.

The expiration date of the orphan drug market exclusivity: October 19, 2025

GB Orphan Drug Number: PLGB 32205/0005 – 0006/OD1

The official guidelines for the proper use of antifungal agents should be considered.

The expiration date of the orphan drug market exclusivity: October 19, 2025

GB Orphan Drug Number: PLGB 32205/0005 – 0006/OD2

Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23

CRYSVITA is suitable for the treatment of X-linked hypophosphatemia in children 1 year and older and adolescents with skeletal growth. Radiological evidence shows bone disease

Orphan drug market exclusivity expiration date: February 21, 2028

GB Orphan Drug Number: PLGB 50262/0001/OD1

Cystadrops are indicated for the treatment of corneal cystine crystal deposits in adults and children over 2 years of age with cystinopathy.

The expiration date of the orphan drug market exclusivity: January 23, 2027

GB Orphan Drug Number: PLGB 15266/0021/OD1

Orphan disease: acute myeloid leukemia

According to the World Health Organization (WHO) classification, Dacogen is suitable for the treatment of newly diagnosed adult patients with newly diagnosed or secondary acute myeloid leukemia (AML) who are not candidates for standard induction chemotherapy.

The expiration date of the orphan drug market exclusivity: September 24, 2024

GB Orphan Drug Number: PLGB 00242/0662/OD1

Orphan disease: plasma cell myeloma

Orphan drug market exclusivity expiration date: May 24, 2026

GB Orphan Drug Number: PLGB 00242/0676 – 0677/OD1

Orphan disease: systemic light chain (AL) amyloidosis

Authorized orphan indication: Darzalex combined with cyclophosphamide, bortezomib and dexamethasone for the treatment of newly diagnosed adult patients with systemic light chain (AL) amyloidosis

The expiration date of the orphan drug market exclusivity: October 11, 2031

GB Orphan Drug Number: PLGB 00242/0677/OD2

Orphan disease: acute myeloid leukemia

Daurismo is used in combination with low-dose cytarabine to treat newly diagnosed adult patients with new or secondary acute myeloid leukemia (AML) who are not suitable for standard induction chemotherapy.

The expiration date of the orphan drug market exclusivity: June 29, 2030

GB Orphan Drug Number: PLGB 00057/1687 – 1688/OD1

Orphan disease: hepatic vein occlusive disease

Hematopoietic stem cell transplantation (HSCT) is used to treat severe hepatic venous occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS).

It is suitable for adults and teenagers, children and babies over 1 month.

The expiration date of the orphan drug market exclusivity: October 22, 2023

GB Orphan Drug Number: PLGB 31626/0005/OD1

(S)-2-nitro-6-(4-(trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b][1,3]oxa Oxazine

Dovprela is used in combination with Bedaquinoline and Linezolid to treat extensively drug-resistant (XDR) or multidrug-resistant (MDR) tuberculosis (TB) that is intolerant or unresponsive in adults.

The expiration date of the orphan drug market exclusivity: August 4, 2030

GB Orphan Drug Number: PLGB 46302/0234/OD1

Epidyolex is indicated for the adjuvant treatment of seizures associated with Lennox Gastaut syndrome (LGS), in combination with clobazam, and for patients 2 years of age and older.

The expiration date of the orphan drug market exclusivity: September 23, 2029

GB Orphan Drug Number: PLGB 36772/0001/OD1

Epidyolex is indicated for the adjuvant treatment of epileptic seizures associated with Dravet syndrome (DS). It is used in combination with clobazam and is suitable for patients 2 years of age and older.

The expiration date of the orphan drug market exclusivity: September 23, 2029

GB Orphan Drug Number: PLGB 36772/0001/OD2

Orphan disease: tuberous sclerosis

Authorized orphan indications: Epidyolex is suitable for the adjuvant treatment of epileptic seizures associated with tuberous sclerosis (TSC) in patients 2 years and older.

The expiration date of the orphan drug market exclusivity: August 5, 2031

GB Orphan Drug Number: PLGB 36772/0001/OD3

Orphan disease: spinal muscular atrophy

Evrysdi is suitable for the treatment of 5q spinal muscular atrophy (SMA) in patients who have been clinically diagnosed as SMA type 1, type 2, or type 3 or have 1 to 4 copies of SMN2 for 2 months and older.

Orphan drug market exclusivity expiration date: May 19, 2031

GB Orphan Drug Number: PLGB 00031/0920/OD1

Orphan disease: treatment of multiple myeloma

Farydak is used in combination with bortezomib and dexamethasone to treat adult patients with relapsed and/or refractory multiple myeloma. These patients have previously received at least two regimens, including bortezomib and an immunomodulator.

Orphan drug market exclusivity expiration date: September 1, 2025

GB Orphan Drug Number: PLGB 53958/0007 – 0009/OD1

Fintepla is suitable for the treatment of seizures associated with Dravet syndrome, as an add-on therapy to other anti-epileptic drugs, and is suitable for patients 2 years and older.

The expiration date of the orphan drug market exclusivity: December 21, 2030

GB Orphan Drug Number: PLGB 45831/0001/OD1

Galafold is suitable for long-term treatment of adults and adolescents aged 16 years and older who are diagnosed with Fabry disease (α-galactosidase A deficiency) and have suitable mutations (see table in section 5.1).

Orphan drug market exclusivity expiration date: May 31, 2026

GB Orphan Drug Number: PLGB 25823/0002/OD1

Orphan disease: chronic lymphocytic leukemia (CLL)

The combination of Gazyvaro and chlorambucil is indicated for the treatment of previously untreated adult patients with CLL, and comorbidities make it unsuitable for treatment based on full-dose fludarabine (see section 5.1).

The expiration date of the orphan drug market exclusivity: July 24, 2024

GB Orphan Drug Number: PLGB 00031/0856/OD1

Orphan disease: follicular lymphoma (FL)

Gazyvaro combined with chemotherapy, followed by Gazyvaro maintenance therapy in patients who reach the response, is suitable for the treatment of previously untreated patients with advanced FL (see section 5.1)

Gazyvaro combined with bendamustine, followed by Gazyvaro maintenance therapy is suitable for the treatment of FL patients who did not respond during treatment with rituximab or rituximab-containing regimens or up to 6 months later. There is progress.

The expiration date of the orphan drug market exclusivity period: June 15, 2026

GB Orphan Drug Number: PLGB 00031/0856/OD2

Orphan disease: acute liver porphyria

A synthetic double-stranded siRNA oligonucleotide targeting δ-aminolevulinic acid synthase 1 mRNA is covalently linked to a ligand containing three N-acetylgalactosamine residues

Treatment of acute hepatic porphyria (AHP) in adults and adolescents 12 years and older

The expiration date of the orphan drug market exclusivity period: March 4, 2030

GB Orphan Drug Number: PLGB 43942/0001/OD1

Part of the appropriate combination treatment plan for MDR-TB for adults and children 28 days and older, because an effective treatment plan cannot be developed due to drug resistance or tolerance.

Official guidelines for the proper use of antimicrobial agents should be considered.

The expiration date of the orphan drug market exclusivity: April 9, 2024

GB Orphan Drug Number: PLGB 35068/0003/OD1

Orphan disease: Hepatitis D virus infection

Treatment of chronic hepatitis D virus (HDV) infection in patients with plasma (or serum) HDV-RNA positive into chronically compensated liver disease.

The expiration date of the orphan drug market exclusivity: August 5, 2030

GB Orphan Drug Number: PLGB 50662/0002/OD1

Orphan status: non-24-hour sleep-wake disorder for blind people without light perception

HETLIOZ is suitable for the treatment of non-24-hour sleep-wake disorders (Non-24) in adults who are completely blind.

Orphan drug market exclusivity expiration date: July 7, 2025

GB Orphan Drug Number: PLGB 43460/0001/OD1

Ex vivo expanded autologous human corneal epithelium containing stem cells

Orphan disease: corneal disease with lack of stem cells in the cornea (limbal) due to eye burns

Treatment of moderate to severe limbal stem cell deficiency (defined as superficial corneal neovascularization in at least two corneal quadrants, central corneal involvement, severe visual impairment), unilateral or bilateral, due to physical or chemical eye burns. A biopsy requires at least 1-2 mm2 of undamaged limbus.

Orphan drug market exclusivity expiration date: February 19, 2025

GB Orphan Drug 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 disease: acute lymphoblastic leukemia

Iclusig is suitable for adult patients with the following diseases

Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph ALL) resistant to dasatinib; patients who are intolerant to dasatinib and subsequent imatinib treatment is not clinically suitable; or people with T315I mutations.

The expiration date of the orphan drug market exclusivity: July 3, 2023

GB Orphan Drug 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 disease: chronic myeloid leukemia

Iclusig is suitable for adult patients with the following diseases

The expiration date of the orphan drug market exclusivity: July 3, 2023

GB Orphan Drug Number: PLGB 49635/0001 – 0003/OD2

Streptococcus pyogenes recombinant IgG degrading enzyme

Orphan status: transplant rejection after solid organ transplantation

Idefirix is suitable for desensitization treatment of highly sensitive adult kidney transplant patients who are positive for cross-matching with available deceased donors. Patients who are unlikely to undergo transplantation under the existing renal distribution system (including a priority plan for highly sensitive patients) should retain the use of Idevirix.

The expiration date of the orphan drug market exclusivity: September 1, 2030

GB Orphan Drug Number: PLGB 46323/0002/OD1

Recombinant fusion protein linking human coagulation factor IX and human albumin

Treatment and prevention of bleeding in patients with hemophilia B (congenital factor IX deficiency).

IDELVION can be used for all ages.

Orphan drug market exclusivity expiration date: May 13, 2026

GB Orphan Drug Number: PLGB 00231/0337 – 0340/OD1

1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one

Orphan disease: chronic lymphocytic leukemia

IMBRUVICA is used as a single agent or in combination with rituximab or obinutuzumab to treat previously untreated adult patients with chronic lymphocytic leukemia (CLL).

IMBRUVICA is used as a single agent or in combination with bendamustine and rituximab (BR) to treat adult CLL patients who have received at least one previous treatment.

The expiration date of the orphan drug market exclusivity: October 23, 2024

GB Orphan Drug Number: PLGB 00242/0687 – 0691/OD1

1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one

Orphan disease: mantle cell lymphoma

IMBRUVICA is suitable as a single agent for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL).

The expiration date of the orphan drug market exclusivity: October 23, 2024

GB Orphan Drug Number: PLGB 00242/0687 – 0691/OD2

1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one

Orphan disease: lymphoplasmacytic lymphoma (also known as Waldenstrom's macroglobulinemia)

IMBRUVICA is suitable as a single agent for the treatment of adult patients with Waldenström's macroglobulinemia (WM) who have received at least one previous treatment, or for the first-line treatment of patients who are not suitable for chemoimmunotherapy. IMBRUVICA is used in combination with rituximab to treat adult patients with WM.

Orphan drug market exclusivity expiration date: July 7, 2025

GB Orphan Drug Number: PLGB 00242/0687 – 0691/OD3

Imnovid in combination with dexamethasone is used to treat adult patients with relapsed and refractory multiple myeloma, these patients have previously received at least two treatment options, including lenalidomide and bortezomib, and in the last treatment Proven disease progression

The expiration date of the orphan drug market exclusivity: August 8, 2023

GB Orphan Drug Number: PLGB 50412/0002 – 0005/OD1

Active substance: fedratinib dihydrochloride monohydrate

Inrebic is suitable for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or primary thrombocythemia myelofibrosis. These patients are Janus-related kinases ( JAK) Inhibitor-naïve or patients who have been treated with Ruxotinib.

The expiration date of the orphan drug market exclusivity: April 16, 2031

GB Orphan Drug Number: PLGB 50412/0022/OD1

Orphan disease: Cushing syndrome

Isturisa is suitable for the treatment of endogenous Cushing syndrome in adults.

The expiration date of the orphan drug market exclusivity: January 13, 2030

GB Orphan Drug Number: PLGB 15266/0029 – 0031/OD1

Jorveza is indicated for the treatment of eosinophilic esophagitis (EoE) in adults (over 18 years of age).

Orphan drug market exclusivity expiration date: January 10, 2028

GB Orphan Drug 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-dimethyl Propoxy)-1H-pyrazole-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide (elexacaftor)

Kaftrio is used in combination with ivacaftor 150 mg tablets to treat cystic fibrosis (CF) in patients 12 years of age and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see section 5.1 ) ).

The expiration date of the orphan drug market exclusivity: August 21, 2030

GB Orphan Drug Number: PLGB 22352/0012/OD1

N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide

Treatment of cystic fibrosis (CF) in patients 6 years and older with a G551D mutation in the CFTR gene

The expiration date of the orphan drug market exclusivity: July 25, 2022

GB Orphan Drug Number: PLGB 22352/0008 – 0011/OD1

N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide

Kalydeco tablets are suitable for use in combination with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults and adolescents aged 12 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1 )

The expiration date of the orphan drug market exclusivity: July 25, 2022

GB Orphan Drug Number: PLGB 22352/0008/OD1

Recombinant human lysosomal acid lipase

Orphan disease: lysosomal acid lipase deficiency

Kanuma is suitable for long-term enzyme replacement therapy (ERT) for patients with lysosomal acid lipase (LAL) deficiency of all ages.

Orphan drug market exclusivity expiration date: September 1, 2025

GB Orphan Drug Number: PLGB 31775/0002/OD1

Active substance: Smeltinib bisulfate

Authorized orphan indication: treatment of symptomatic, inoperable plexiform neurofibroma (PN) in pediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and older.

The expiration date of the orphan drug market exclusivity: August 8, 2031

GB Orphan Drug Number: PLGB 17901/0356-0357/OD1

Orphan disease: Cushing syndrome

Ketoconazole HRA is indicated for the treatment of endogenous Cushing syndrome in adults and adolescents over 12 years of age.

Orphan drug market exclusivity expiration date: November 21, 2024

GB Orphan Drug Number: PLGB 51757/0002/OD1

Autologous T cells transduced with a lentiviral vector containing a chimeric antigen receptor for CD19

Kymriah is suitable for the treatment of children and young adults with B-cell acute lymphoblastic leukemia (ALL) under 25 years of age who are refractory, relapsed after transplantation, or relapsed for the second time or later.

Orphan drug market exclusivity expiration date: August 27, 2028

GB Orphan Drug Number: PLGB 00101/1101/OD1

Active substance: tisagenlecleucel autologous T cell transduction lentiviral vector, which contains a chimeric antigen receptor for CD19

Orphan disease: diffuse large B-cell lymphoma

Kymriah is suitable for the treatment of:

Orphan drug market exclusivity expiration date: August 27, 2028

GB Orphan Drug Number: PLGB 00101/1101/OD2

Kyprolis is used in combination with daratumomab and dexamethasone, lenalidomide and dexamethasone or dexamethasone alone for the treatment of adult patients with multiple myeloma who have received at least one previous treatment.

Orphan drug market exclusivity expiration date: November 23, 2025

GB Orphan Drug Number: PLGB 13832/0023 – 0025/OD1

Enzyme replacement therapy is used to treat non-neurological manifestations in patients with mild to moderate alpha mannoside poisoning. See sections 4.4 and 5.1.

Orphan drug market exclusivity expiration date: March 27, 2028

GB Orphan Drug Number: PLGB 08829/0188/OD1

Orphan disease: skin T-cell lymphoma

Ledaga is suitable for the local treatment of mycosis fungoides cutaneous T-cell lymphoma (MF CTCL) in adult patients

Orphan drug market exclusive period expiration date: March 7, 2027 GB Orphan drug number: PLGB 15720/0005/OD1

Active substance: Autologous CD34 cell-rich clusters, including hematopoietic stem cells and progenitor cells transduced in vitro with a lentiviral vector encoding human arylsulfatase A gene

Libmeldy is used to treat metachromatic leukodystrophy (MLD), which is characterized by a biallelic mutation in the arylsulfatase A (ARSA) gene that leads to a decrease in ARSA enzyme activity:

The expiration date of the orphan drug market exclusivity: December 18, 2030

GB Orphan Drug 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-cysteyl-L- Tyrosyl-D-tryptophan-L-lysyl-L-threonyl

Orphan disease: gastrointestinal pancreatic neuroendocrine tumors

Lutathera is suitable for the treatment of unresectable or metastatic, progressive, well-differentiated (G1 and G2), somatostatin receptor-positive gastrointestinal pancreatic neuroendocrine tumors (GEP NET) in adults.

The expiration date of the orphan drug market exclusivity: September 28, 2027

GB Orphan Drug Number: PLGB 35145/0003/OD1

Adeno-associated virus vector serotype 2 containing human RPE65 gene

Orphan disease: hereditary retinal dystrophy (originally named Leber congenital amaurosis)

Luxturna is suitable for the treatment of adult and child patients with vision loss caused by hereditary retinal dystrophy caused by confirmed biallelic RPE65 mutations, and these patients have enough viable retinal cells.

The expiration date of the orphan drug market exclusivity: December 5, 2028

GB Orphan Drug Number: PLGB 00101/1104/OD1

Adeno-associated virus vector serotype 2 containing human RPE65 gene

Orphan disease: hereditary retinal dystrophy (originally named retinitis pigmentosa)

The expiration date of the orphan drug market exclusivity: December 5, 2028

GB Orphan Drug Number: PLGB 00101/1104/OD2

Orphan disease: Mucopolysaccharidosis type VII (Sly syndrome)

Mepsevii is suitable for the treatment of non-neurological manifestations of mucopolysaccharidosis VII (MPS VII; Sly syndrome).

Orphan drug market exclusivity expiration date: August 27, 2028

GB Orphan Drug Number: PLGB 474793/0002/OD1

Orphan disease: familial partial lipodystrophy

Myalepta can be used as an adjunct to diet, as an alternative therapy for the treatment of complications of leptin deficiency in patients with lipodystrophy (LD):

Orphan drug market exclusivity expiration date: August 1, 2028

GB Orphan Drug Number: PLGB 50688/0008-0010/OD1

Myalepta can be used as an adjunct to diet, as an alternative therapy for the treatment of complications of leptin deficiency in patients with lipodystrophy (LD):

Adults and children 12 years of age and older with acquired partial LD (Barraquer-Simons syndrome) fail to achieve adequate metabolic control with standard treatment.

Orphan drug market exclusivity expiration date: August 1, 2028

GB Orphan Drug Number: PLGB 50688/0008-0010/OD2

Myalepta can be used as an adjunct to diet and as an alternative therapy for the treatment of complications of leptin deficiency in patients with lipodystrophy (LD): adults and children 2 years and older with acquired systemic LD (Lawrence syndrome)

Orphan drug market exclusivity expiration date: August 1, 2028

GB Orphan Drug Number: PLGB 50688/0008-0010/OD3

Myalepta can be used as an adjunct to diet and as an alternative therapy for the treatment of complications of leptin deficiency in patients with lipodystrophy (LD): diagnosed with congenital systemic LD (Berardinelli-Seip syndrome)

Orphan drug market exclusivity expiration date: August 1, 2028

GB Orphan Drug Number: PLGB 50688/0008-0010/OD4

Orphan disease: acute myeloid leukemia

MYLOTARG is suitable for combined therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of newly untreated CD33-positive acute myeloid leukemia (AML) patients aged 15 years and older, acute Except for promyelocytic leukemia (APL).

The expiration date of the orphan drug market exclusivity: April 23, 2028

GB Orphan Drug Number: PLGB 00057/1591/OD1

Namuscla is suitable for the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonia

The expiration date of the orphan drug market exclusivity: December 20, 2028

GB Orphan Drug Number: PLGB 35507/0200/OD1

Natpar is suitable for the adjuvant treatment of adult patients with chronic hypoparathyroidism that cannot be adequately controlled by standard treatment alone.

The expiration date of the orphan drug market exclusivity: April 26, 2027

GB Orphan Drug Number: PLGB 16189/0067 – 0070/OD1

Orphan disease: follicular thyroid cancer

NEXAVAR is suitable for the treatment of patients with progressive, locally advanced or metastatically differentiated (follicular/Hürthle cell) thyroid cancer refractory to radioactive iodine.

Orphan drug market exclusivity expiration date: May 27, 2024

GB Orphan Drug Number: PLGB 00010/0701/OD1

Orphan disease: papillary thyroid carcinoma

NEXAVAR is suitable for the treatment of patients with progressive, locally advanced or metastatic differentiated (papillary) thyroid cancer refractory to radioactive iodine.

Orphan drug market exclusivity expiration date: May 27, 2024

GB Orphan Drug Number: PLGB 00010/0701/OD2

Active substance: Proteolytic enzyme concentrate rich in bromelain

Orphan disease: partial deep skin burns and full-thickness burns

NexoBrid is suitable for removing eschar from adults with deep partial and full-thickness thermal burns.

The expiration date of the orphan drug market exclusivity: December 20, 2022

GB Orphan Drug Number: PLGB 43948/0002 – 0003/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 is used in combination with lenalidomide and dexamethasone to treat adult patients with multiple myeloma who have received at least one previous treatment.

The expiration date of the orphan drug market exclusivity: November 23, 2026

GB Orphan Drug Number: PLGB 16189/0109 – 0111/OD1

Obiltoxaximab SFL is suitable for the treatment of inhaled anthrax caused by Bacillus anthracis in combination with appropriate antibacterial drugs for all age groups (see section 5.1).

When alternative therapies are inappropriate or unavailable, Obiltoxaximab SFL is suitable for post-exposure prevention of inhaled anthrax in all age groups (see section 5.1).

The expiration date of the orphan drug market exclusivity: November 19, 2030

GB Orphan Drug Number: PLGB 54280/0001/OD1

Orphan disease: primary biliary cirrhosis

Ocaliva is used in combination with ursodeoxycholic acid (UDCA) to treat primary biliary cholangitis (PCB) (also known as primary biliary cirrhosis) in adults who do not respond well to UDCA, or as a single-agent treatment Adults who tolerate UDCA.

The expiration date of the orphan drug market exclusivity: December 15, 2026

GB Orphan Drug Number: PLGB 48025/0002 – 0003/OD1

Combination with 5-fluorouracil (5 FU) and leucovorin (LV) to treat pancreatic metastatic adenocarcinoma in adult patients who have progressed after receiving gemcitabine.

Orphan drug market exclusivity expiration date: October 18, 2026

GB Orphan Drug Number: PLGB 05815/0111/OD1

Synthetic double-stranded siRNA oligonucleotide targeting 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 drug market exclusivity expiration date: August 29, 2028

GB Orphan Drug Number: PLGB 50597/0002/OD1

Orphan disease: pulmonary hypertension

Opsumit, as a monotherapy or combination therapy, is suitable for long-term treatment of pulmonary hypertension (PAH) in adult patients with WHO functional class (FC) II to III.

Efficacy has been shown in the PAH population, including idiopathic and hereditary PAHs, PAHs associated with connective tissue diseases, and PAHs associated with corrected simple congenital heart disease.

Orphan drug market exclusivity expiration date: December 27, 2023

GB Orphan Drug Number: PLGB 00242/0663/OD1

Orphan disease: a congenital error in the synthesis of primary bile acids

Treatment of primary bile acids in infants, children and adolescents and adults from 1 month to 18 years of age caused by 3β-hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ43-oxysteroid-5β-reductase deficiency Synthetic congenital errors.

The expiration date of the orphan drug market exclusivity: September 16, 2023

GB Orphan Drug Number: PLGB 44776/0002 – 0003/OD1

Authorized orphan indications: treatment of moderate (persistent epithelial defect) or severe (corneal ulcer) neurotrophic keratitis in adults.

Orphan drug market exclusive date: July 10, 2027

GB Orphan Drug Number: PLGB 32162/0002/OD1

A synthetic double-stranded siRNA oligonucleotide targeting hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues

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

The expiration date of the orphan drug market exclusivity: November 23, 2030

GB Orphan Drug Number: PLGB 43942/0003/OD1

Palynziq is indicated for the treatment of phenylketonuria (PKU) patients 16 years of age and older who have insufficient blood phenylalanine control (blood phenylalanine levels greater than 600 micromoles/liter), although there are previous treatments available choose.

Orphan drug market exclusive date: May 8, 2029

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

Orphan disease: biliary tract cancer

Authorized orphan indication: For the treatment of adults with locally advanced or metastatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement, and has progressed after at least one previous systemic treatment.

The expiration date of the orphan drug market exclusivity: April 7, 2031

GB Orphan Drug Number: PLGB 42338/0008-0010/OD1

Treatment of adult hypoadrenal function.

Orphan drug market exclusivity expiration date: November 14, 2021

GB Orphan Drug Number: PLGB 39534/0009-0010/OD1

Orphan disease: diffuse large B-cell lymphoma

Polivy is used in combination with bendamustine and rituximab to treat adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are not suitable for hematopoietic stem cell transplantation.

The expiration date of the orphan drug market exclusivity: January 20, 2030

GB Orphan Drug Number: PLGB 00031/0912/OD1

Orphan disease: skin 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 previous systemic treatment.

Orphan drug market exclusivity expiration date: November 26, 2028

GB Orphan Drug 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 disease: cell-mediated reactivation of cytomegalovirus disease in immunocompromised patients

PREVYMIS is suitable for the prevention of cytomegalovirus (CMV) reactivation and disease in adult CMV seropositive recipients [R] of allogeneic hematopoietic stem cell transplantation (HSCT).

Official guidelines for the appropriate use of antiviral drugs should be considered.

Orphan drug market exclusivity expiration date: January 10, 2028

GB Orphan Drug Number: PLGB 53095/0046 – 0048/OD1

Active substance: Thiamine (cysteamine bitartrate)

PROCYSBI is suitable for the treatment of proven nephrotic cystinopathy. Cysteamine reduces the accumulation of cystine in certain cells (such as white blood cells, muscles, and liver cells) in patients with nephrotic cystinopathy, and when treatment is started early, it delays the development of renal failure.

Orphan drug market exclusivity expiration date: September 10, 2023

GB Orphan Drug Number: PLGB 08829/0190 – 0191/OD1

Chimeric monoclonal antibody against GD2

Qarziba is used for the treatment of high-risk neuroblastoma patients of 12 months and older who have previously received induction chemotherapy and achieved at least partial remission, followed by myeloablization and stem cell transplantation, as well as patients with a history of relapse. Or refractory neuroblastoma, with or without residual disease. Before treating recurrent neuroblastoma, other appropriate measures should be taken to stabilize any actively progressing disease.

For patients with a history of relapsed/refractory disease and patients who have not achieved complete remission after first-line treatment, Qarziba should be combined with interleukin-2 (IL-2).

Orphan drug market exclusivity expiration date: May 11, 2027

GB Orphan Drug Number: PLGB 44185/0005/OD1

Orphan disease: carbamoyl phosphate synthase 1 deficiency

Treatment of carbamoyl phosphate synthase 1 deficiency:

RAVICTI must be used in conjunction with dietary protein restriction, and in some cases, dietary supplements (for example, essential amino acids, arginine, citrulline, protein-free calorie supplements) must be used.