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Vertex Pharmaceuticals Orkambi sNDA PDUFA

09/30/2016 - 00:00

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U.S. FDA Accepts for Priority Review Supplemental New Drug Application for the Use of ORKAMBIĀ® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6 to 11 who have Two Copies of the F508del Mutation
-Approximately 2,400 children ages 6 to 11 have two copies of the F508del mutation in the U.S.-

-Target review date of September 30, 2016 set for the FDA's decision on the application-

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for the use of ORKAMBIĀ® (lumacaftor/ivacaftor) in people with cystic fibrosis (CF) ages 6 to 11 who have two copies of the F508del mutation. The FDA granted Vertex's request for Priority Review of this sNDA, and a target review date of September 30, 2016 was set under the Prescription Drug User Fee Act (PDUFA).

"As complications related to CF can occur early in life, we believe it is important to begin treatment as early as possible and this supplemental New Drug Application for approval of ORKAMBI in children as young as six is an important step in that direction," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "This submission demonstrates Vertex's continued progress toward our goal of developing medicines for all people with CF."

The sNDA is based on data from an open label Phase 3 clinical safety study of ORKAMBI. Data from this study will be presented at the 39th European Cystic Fibrosis Society conference being held June 8 to 11 in Basel, Switzerland.

To support potential approval in the European Union, a six-month Phase 3 efficacy study of children ages 6 to 11 is ongoing. Vertex recently completed enrollment in this study and, pending data from the study, plans to submit a Marketing Authorization Application (MAA) variation in the European Union in the first half of 2017 for children ages 6 to 11 who have two copies of the F508del mutation. The primary endpoint of this efficacy study is the absolute change in lung clearance index (LCI). There are approximately 3,400 children ages 6 to 11 who have two copies of the F508del mutation in the European Union.
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Source: press release, 5/31/16. http://investors.vrtx.com/releasedetail.cfm?ReleaseID=973361

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