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List of the latest marketing authorisations and orphan medicinal products designations
Detailed information on European orphan medicinal products designation applications is available on the EMA website. A full list of designated and authorised orphan medicinal products in Europe available at: ec.europa.eu.
Learn more about the Orphan designation process in Europe
Recent marketing authorisations
See a list of orphan medicines currently authorised in the EU.
|
Tavneos® (avacopan) |
Vifor Fresenius Medical Care Renal Pharma France | Adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) | 11/01/2022 |
|
Voraxaze® (glucarpidase) |
SERB SAS | Adults and children (aged 28 days and older) with delayed methotrexate elimination or at risk of methotrexate toxicity | 11/01/2022 |
|
Lonapegsomatropin Ascendis Pharma® (lonapegsomatropin) |
Ascendis Pharma Endocrinology Division A/S | Children who do not produce enough growth hormone (GHD) | 11/01/2022 |
|
Oxbryta® (voxelotor) |
Global Blood Therapeutics Netherlands B. V. |
Haemolytic anaemia, and +12 years old sickle cell disease |
14/02/2022 |
|
Ngenla® (somatrogon) |
Pfizer Europe MA EEIG |
Children and adolescents with growth hormone deficiency |
14/02/2022 |
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The voice of rare disease patients in Europe
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiative
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiative