Rare disease (orphan) designations

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This page allows you to find information on rare disease (orphan) designations based on applications that have been assessed by the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP).

An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market. Applications for orphan designation are examined by the COMP, which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.

For more information, see Orphan designation.

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EPAR Search results
Active substance Disease / condition Date of decision Decision Medicine name

Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene

Treatment of glycogen storage disease type II (Pompe's disease) 2018-04-16 Positive  

adeno-associated viral vector serotype 9 encoding miRNA against human superoxide dismutase 1

Treatment of amyotrophic lateral sclerosis 2018-04-16 Positive  

autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo

Treatment of ovarian cancer 2018-04-16 Positive  

branaplam

Treatment of spinal muscular atrophy 2018-04-16 Positive  

burosumab

Treatment of phosphaturic mesenchymal tumour 2018-04-16 Positive  

genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII

Treatment of epidermolysis bullosa 2018-04-16 Positive  

polatuzumab vedotin

Treatment of diffuse large B-cell lymphoma 2018-04-16 Positive  

(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide

Treatment of C3 glomerulopathy 2018-03-21 Positive  

Dimethyl fumarate

Treatment of Friedreich's ataxia 2018-03-21 Positive  

Docosahexaenoic acid ethyl ester

Treatment of sickle cell disease 2018-03-21 Positive  

Efgartigimod alfa

Treatment of myasthenia gravis 2018-03-21 Positive  

Gemfibrozil

Treatment of neuronal ceroid lipofuscinosis 2018-03-21 Positive  

Ivosidenib

Treatment of biliary tract cancer 2018-03-21 Positive  

Larotrectinib

Treatment of salivary gland cancer 2018-03-21 Positive  

Melatonin

Treatment of neonatal encephalopathy 2018-03-21 Positive  

Miransertib

Treatment of Proteus syndrome 2018-03-21 Positive  

Patidegib

Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome) 2018-03-21 Positive  

Recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA

Treatment of haemophilia B 2018-03-21 Positive  

Recombinant human acid alpha-glucosidase

Treatment of glycogen storage disease type II (Pompe's disease) 2018-03-21 Positive  

Recombinant modified ricin toxin A-chain subunit

Prevention of ricin poisoning 2018-03-21 Positive  

Ribavirin

Treatment of Crimean-Congo haemorrhagic fever 2018-03-21 Positive  

Tazemetostat

Treatment of diffuse large B-cell lymphoma 2018-03-21 Positive  

Tazemetostat

Treatment of follicular lymphoma 2018-03-21 Positive  

Tazemetostat

Treatment of malignant mesothelioma 2018-03-21 Positive  

Melatonin

Treatment of partial deep dermal and full thickness burns 2018-03-13 Negative