FDA Grants Priority Review For Actelion Miglustat

FDA has accepted Actelion’s 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).

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.

Actelion has been informed by the FDA that the sNDA will be reviewed by the Endocrine and Metabolic Drug Advisory Committee (EMDAC) on 12th January 2010.

Jean-Paul Clozel, MD and chief executive officer, said: “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.”

In 2008, FDA granted orphan drug status to Miglustat for NP-C in the US.