Actelion receives FDA complete response letter for Zavesca (miglustat) for the treatment of Niemann-Pick type C disease
Actelion Ltd announced that the company has received a complete response letter from the U.S. food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for Zavesca® (miglustat) for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C (NP-C) disease, a rare, neurodegenerative genetic disorder.
The FDA has requested additional preclinical and clinical information. Actelion will work diligently with the FDA to explore the best ways to address the points raised by the agency.
Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "We remain committed to bringing an approved treatment to patients suffering from NP-C disease and in this spirit we will continue the dialogue with the FDA."
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