Actelion receives positive FDA opinion of Zavesca

Actelion has announced positive Food and Drug Administration (FDA) approval of its Niemann-Pick type C (NP-C) therapy Zavesca (miglustat).

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted ten in favour and three objections to approving the drug after taking its benefit to risk profile into consideration.

Its use is being considered for the treatment of progressive neurological manifestations in both adult and paediatric patients with NP-C.

The neurodegenerative genetic disorder is a very rare, progressive and eventually fatal disease, brought about by the build up of glycosphingolipids.

Jean-Paul Clozel, chief executive officer of Actelion, stated: “We are pleased that the Advisory Committee today recognised the importance of this therapeutic advance and voted to recommend approval of the supplemental New Drug Application.”

In other company news, Actelion announced in December that its dual orexin receptor antagonist almorexant met both primary and secondary endpoints during a trial measuring its capabilities.