- EMA Orphan Designations
- European Medicines Agency Orphan Designations
- European Medicines Agency
- EMA Orphan
- Rare Disease Designations
EMA Rare Disease Designations
The European Medicines Agency (EMA) Rare Disease Designations dataset includes 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).
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About 30 million people living in the European Union (EU) suffer from a rare disease. The European Medicines Agency (EMA) plays a central role in facilitating the development and authorization of medicines for rare diseases, which are termed ‘orphan medicines’ in the medical world.
Developing medicines intended for small numbers of patients have a little commercial incentive under normal market conditions. Therefore, the EU offers a range of incentives to encourage the development of designated orphan medicines.
The Committee for Orphan Medicinal Products (COMP) is responsible for reviewing applications for orphan designation for medicines that are intended for the diagnosis, prevention or treatment of rare diseases.
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.
About this Dataset
European Economic Area Countries
John Snow Labs; European Medicines Agency (EMA);
|Source License URL|
|Source License Requirements||
European Medicines Agency, EMA Orphan, Rare Disease Designations
EMA Orphan Designations, European Medicines Agency Orphan Designations
|Active_Substance||Active substance of medicinal product||string||-|
|Disease_Condition||Conditions of the rare disease||string||-|
|Date_Of_Decision||COMP decision date||date||-|
|Decision_Type||COMP decision type||string||-|
|Medicine_Name||Orphan drug name||string||-|
|Agency_Product_Number||The agency product number||string||-|
|EU_Designation_Number||European designation number||string||-|
|First_Published||Date of first publication||date||-|
|Revision_Date||Date of revision||date||-|
|EMA_Link||URL of the product at EMA website||string||-|
|Active Substance||Disease Condition||Date Of Decision||Decision Type||Medicine Name||Agency Product Number||EU Designation Number||First Published||Revision Date||EMA Link|
|Caffeine citrate||Prevention of bronchopulmonary dysplasia||2014-04-11||Positive||Blectifor||EMEA/H/C/004100||EU/3/14/1261||2014-06-05||2014-06-05||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3141261|
|Vatreptacog alfa (activated)||Treatment of haemophilia B||2012-08-09||Withdrawn||EU/3/12/1032||2012-09-27||2015-03-06||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3121032|
|unoprostone isopropyl||Treatment of retinitis pigmentosa||2013-06-19||Withdrawn||EU/3/13/1146||2013-07-11||2016-08-11||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3131146|
|Pyridoxal 5'-phosphate||Treatment of pyridoxamine 5'-phosphate oxidase deficiency||2014-10-15||Positive||EU/3/14/1347||2014-11-12||2014-11-12||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3141347|
|Norursodeoxycholic acid||Treatment of primary sclerosing cholangitis||2014-07-04||Positive||EU/3/14/1288||2014-07-16||2014-07-16||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3141288|
|2-(2-Methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide||Treatment of small cell lung cancer||2014-08-22||Positive||EU/3/14/1324||2014-10-02||2014-10-02||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3141324|
|Argon||Treatment of perinatal asphyxia||2018-06-27||Positive||EU/3/18/2031||2018-08-22||2018-08-22||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182031|
|20-pentaerythritol poly (oxy-1,2-ethanediyl)-carboxymethyl-glycinate-7-ethyl-10-hydroxycamptothecine 10-[1,4'-bipiperidine]-1'-carboxylate||Treatment of ovarian cancer||2011-09-27||Positive||EU/3/11/900||2011-10-17||2011-10-17||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu311900|
|Plitidepsin||Treatment of post-essential thrombocythaemia myelofibrosis||2011-02-23||Withdrawn||EU/3/10/838||2011-03-07||2011-09-06||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu310838|
|Autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 - glutathione S transferase) coupled to oxidised polymannose||Treatment of ovarian cancer||2010-09-20||Positive||EU/3/10/776||2010-10-18||2013-10-17||https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu310776|