1 April 2016 - The EMA has recommended granting a marketing authorisation in the European Union for Galafold (migalastat hydrochloride) for the treatment of Fabry disease, a rare genetic disorder.

Because Fabry disease is rare, Galafold was designated as an orphan medicine by the Committee for Orphan Medicinal Products. Orphan designation gives medicine developers access to incentives such as fee reductions for scientific advice, or the possibility to obtain 10 years’ market exclusivity for an authorised orphan-designated medicine. It is a key instrument available in the EU to encourage the development of medicines for patients with rare diseases. The applicant also received scientific advice on quality, non-clinical and clinical aspects of the application.

For more details, go to: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/04/news_detail_002502.jsp&mid=WC0b01ac058004d5c1