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

(6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid

Treatment of systemic sclerosis 2017-01-12 Positive -

3-pentylbenzeneacetic acid sodium salt

Treatment of Alström syndrome 2017-01-12 Positive -

3-{[2,3,5,6-tetrafluoro-3'-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid

Treatment of non-infectious uveitis 2015-06-19 Positive -

5-aminolevulinic acid

Treatment of glioma 2017-01-12 Positive -

Adeno-associated viral vector containing the human factor IX gene

Treatment of haemophilia B 2015-06-19 Withdrawn -

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

Treatment of spinal muscular atrophy 2015-06-19 Positive -

Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter

Treatment of Wilson's disease 2017-08-23 Positive -

Allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells

Prevention of bronchopulmonary dysplasia 2015-06-19 Positive -

Antisense oligonucleotide directed against TGF-β2 mRNA

Prevention of scarring post glaucoma filtration surgery 2015-06-19 Positive -

Antisense oligonucleotide targeting exon 13 in the USH2A gene

Treatment of retinitis pigmentosa 2017-08-23 Positive -


Treatment of pancreatic cancer 2017-01-12 Positive -


Treatment of myelodysplastic syndromes 2017-08-23 Positive -

Autologous dendritic cells incubated ex vivo with zebularine and factor VIII

Treatment of haemophilia A 2017-01-12 Positive -

Doxorubicin hydrochloride in a lipid-based pegylated nanoparticle modified with a 31-aminoacid peptide targeting nucleolin

Treatment of malignant mesothelioma 2017-01-12 Positive -


Treatment of amyotrophic lateral sclerosis 2015-06-19 Positive -

Fluticasone propionate

Treatment of eosinophilic oesophagitis 2017-01-12 Positive -

Genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1

Treatment of oculopharyngeal muscular dystrophy 2017-01-12 Positive -

Human donor haematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein fused to MYC transcription factor

Treatment in haematopoietic stem cell transplantation 2017-01-12 Positive -

Human hepatoma cell line HepaRG in bioartificial liver

Treatment of acute liver failure 2017-01-12 Positive -

Humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor

Treatment of medulloblastoma 2017-01-12 Positive -


Treatment of antiphospholipid syndrome 2017-01-12 Positive -


Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome) 2017-08-23 Positive -

Leuprorelin acetate

Treatment of congenital hypogonadotropic hypogonadism 2017-01-12 Positive -


Treatment of hepatocellular carcinoma 2017-08-23 Positive -


Treatment of marginal zone lymphoma 2015-06-19 Withdrawn -