European Medicines Agency - News and press release archive - Agency publishes first review of orphan designation

Agency publishes first review of orphan designation

Agency publishes first review of orphan designation
17/09/2010 - The Agency has published the first of its ‘review of orphan designation’ documents. These documents summarise the review of the orphan designation carried out by the Committee for Orphan Medicinal Products (COMP) whenever an orphan medicine reaches marketing authorisation. The review is carried out to check that the criteria underpinning the medicine’s orphan designation still apply.

The publication of these documents has been introduced in order to increase transparency over the Agency’s orphan designation process. The documents summarise the COMP’s position on whether the orphan designation for a medicinal product that is receiving marketing authorisation should be maintained or revoked, and include a discussion on the justification of benefit over other authorised treatments.

The first review document concerns Vpriv (velaglucerase alfa), which was authorised for the treatment of Gaucher disease on 26 August 2010. The COMP concluded that Vpriv’s orphan designation can be maintained on 8 July 2010.

The Agency will continue to publish these documents for all orphan medicinal products at the time of marketing authorisation, and for all extensions of indication approved for orphan medicines already on the market.
European Medicines Agency - News and press release archive - Agency publishes first review of orphan designation