The European Medicines Agency (EMA) hosts a number of websites and systems to support its work. You can browse a selection of these below, A-Z.
Several EMA IT systems will be partially or fully inaccessible at different intervals between 11 and 17 April 2024 due to data transfer operations. For more information, read our news announcement. We apologise for any inconvenience caused. EMA's …
Overview This medicine was designated as an orphan medicine for the treatment of spinocerebellar ataxia in the European Union on 11 November 2022. This means that the developer will receive scientific and regulatory support from EMA to advance their …
At the time of designation, amyotrophic lateral sclerosis affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 52,000 people*, and is below the ceiling for orphan designation, which is 5 people in …
This medicine, also known as ASC618, is made of a virus that has been modified to contain a gene that encodes for factor VIII, the protein that is lacking in patients with haemophilia A. This protein is necessary for the formation of blood clots. When …
At the time of designation, microvillus inclusion disease affected less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 people*, and is below the ceiling for orphan designation, which is 5 people in …
At the time of designation, Leber's hereditary optic neuropathy affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 46,000 people, and is below the ceiling for orphan designation, which is 5 …
Overview This medicine was designated as an orphan medicine for the treatment of pulmonary arterial hypertension (PAH) in the European Union on 21 June 2021. This means that the developer will receive scientific and regulatory support from EMA to advance …
Cystinosis is caused by a defect in the gene for cystinosin, a protein essential for normal cell function. The medicine, also known as AVR-RD-O4, is a gene therapy, produced from immature blood (stem) cells collected from the patient. A therapeutic gene ( …
At the time of designation, perinatal asphyxia affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 51,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This …
Overview This medicine was designated as an orphan medicine for the treatment of invasive Scopulariopsis in the European Union on 14 January 2022. This means that the developer will receive scientific and regulatory support from EMA to advance their …
At the time of designation, DMD affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to fewer than 26,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the …
Overview This medicine was designated as an orphan medicine for the treatment of Krabbe disease in the European Union on 15 October 2021. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to …
At the time of designation, soft tissue sarcoma affected approximately 4.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 238,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. …
Overview On 17 October 2019, orphan designation EU/3/19/2203 was granted by the European Commission to MDC RegAffairs GmbH, Germany, for (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5 …
At the time of designation, fragile X syndrome affected approximately 2.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 113,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. …
At the time of designation, approximately 0.7 in 10,000 people received HSCT every year in the European Union (EU). This was equivalent to a total of around 36,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This …
Overview This medicine was designated as an orphan medicine for the prevention of bronchopulmonary dysplasia in the European Union on 14 January 2022. This means that the developer will receive scientific and regulatory support from EMA to advance their …
CHARGE syndrome is a genetic disorder often caused by mutations (changes) in the genes for a protein called CHD7 that is needed for normal development of an unborn child. Patients with CHARGE syndrome may have an eye defect called coloboma, where part of …
The effects of the medicine have been evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with DiGeorge syndrome who have a non-functional thymus were ongoing …
07 days 18:27:22
