Zavesca receives positive vote from FDA for the treatment of Niemann-Pick type C disease

15-Jan-2010 - Switzerland

Actelion Ltd announced that United States (US) Food and Drug Administration's (FDA) Endocrinologic and Metabolic Drugs Advisory Committee voted (10 yes to 3 no) in its final vote in question that the benefit/risk profile of Zavesca® (miglustat) supports its approval for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C (NP-C) disease.

The decision was based on results from the clinical trial OGT 918-007 and two multicenter NP-C disease cohort studies as well as other clinical trials in related lysosomal storage disorders for the safety and tolerability evaluation.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "We are pleased that the Advisory Committee today recognized the importance of this therapeutic advance and voted to recommend approval of the supplemental New Drug Application (sNDA) of Zavesca® for the treatment of NP-C disease. We will continue to work closely with the FDA to facilitate the completion of the priority review of the sNDA."

Zavesca® is the only specific treatment available for patients with NP-C disease. It received approval in the European Union (EU) and other countries in 2009. Zavesca® is also indicated in the US, the EU and other countries for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is unsuitable or is not a therapeutic option. The use of Zavesca® is supported by over 10 years of clinical trials and post-marketing experience across indications.

https://www.bionity.com/en/news/111820/zavesca-receives-positive-vote-from-fda-for-the-treatment-of-niemann-pick-type-c-disease.html