EMA Rare Disease Designations

$179 / year

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).

Complexity

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.

Date Created

2011

Last Modified

2020-07-20

Version

2020-07-20

Update Frequency

Irregular

Temporal Coverage

N/A

Spatial Coverage

European Economic Area Countries

Source

John Snow Labs; European Medicines Agency (EMA);

Source License URL

Source License Requirements

N/A

Source Citation

N/A

Keywords

European Medicines Agency, EMA Orphan, Rare Disease Designations

Other Titles

EMA Orphan Designations, European Medicines Agency Orphan Designations

NameDescriptionTypeConstraints
Active_SubstanceActive substance of medicinal productstring-
Disease_ConditionConditions of the rare diseasestring-
Date_Of_DecisionCOMP decision datedate-
Decision_TypeCOMP decision typestring-
Medicine_NameOrphan drug namestring-
Agency_Product_NumberThe agency product numberstring-
EU_Designation_NumberEuropean designation numberstring-
First_PublishedDate of first publicationdate-
Revision_DateDate of revisiondate-
EMA_LinkURL of the product at EMA websitestring-
Active SubstanceDisease ConditionDate Of DecisionDecision TypeMedicine NameAgency Product NumberEU Designation NumberFirst PublishedRevision DateEMA Link
ArgonTreatment of perinatal asphyxia2018-06-27PositiveEU/3/18/20312018-08-222018-08-22https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182031
Heterologous human adult liver-derived stem cellsTreatment of ornithine transcarbamylase deficiency2011-09-27PositiveEU/3/11/9042011-10-172011-10-17https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu311904
Sodium phenylbutyrateTreatment of ornithine-transcarbamylase deficiency2012-02-09WithdrawnEU/3/12/9502012-03-012013-10-09https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu312950
Recombinant human beta-glucuronidase (vestronidase alfa)Treatment of mucopolysaccharidosis type VII (Sly syndrome)2012-03-21PositiveMepsevii,EMEA/H/C/004438EU/3/12/9732012-04-192018-09-07https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu312973
Pegylated recombinant factor VIIaTreatment of haemophilia B2008-06-03WithdrawnEU/3/08/5522008-08-182015-03-06https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu308552
Monoclonal antibody to human interleukin-6Treatment of post-transplantation lymphoproliferative disorders2002-12-18WithdrawnEU/3/02/1252008-08-182010-02-22https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu302125
humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domainPrevention of graft-versus-host disease2015-11-11PositiveEU/3/15/15752016-01-062018-06-13https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3151575
Adeno-associated viral vector serotype 8 containing the human AIPL1 geneTreatment of Leber's congenital amaurosis type 42011-12-09WithdrawnEU/3/11/9292011-12-202015-03-06https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu311929
ElotuzumabTreatment of multiple myeloma2012-08-09WithdrawnEU/3/12/10372012-09-262016-05-23https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3121037
2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acidTreatment of systemic sclerosis2013-03-12PositiveEU/3/13/11082013-04-052013-04-05https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3131108