US Food and Drug Administration grants priority review for Actelion's miglustat in Niemann-Pick type C disease

19-Nov-2009 - USA

Actelion Ltd announced that a supplemental new drug application (sNDA) for an extension of indication for Zavesca® (miglustat) for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C disease (NP-C) has been accepted by the U.S. food and Drug Administration (FDA).

In the US, Zavesca® is currently indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease where enzyme replacement therapy is unsuitable or is not a therapeutic option.

The sNDA, based on results from the clinical trial OGT 918-007, and two multicenter retrospective cohort studies in patients with NP-C, has been granted a priority review. A priority review designation is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. It also means that the FDA will aim to complete the review within 6 months. Actelion has been informed by the FDA that this sNDA will be reviewed by the Endocrine and Metabolic Drug Advisory Committee (EMDAC) on 12th January 2010.

Jean-Paul Clozel, M.D. and Chief Executive Officer commented: "Actelion is working closely with the FDA to provide information as needed to support the review process and make miglustat available in the United States to patients suffering from this fatal neurodegenerative genetic disorder affecting both children and adults. Miglustat could become the first treatment for NP-C in the USA, which would represent a major therapeutic breakthrough for patients and their treating physicians".

https://www.bionity.com/en/news/109707/us-food-and-drug-administration-grants-priority-review-for-actelion-s-miglustat-in-niemann-pick-type-c-disease.html