PDUFA Date for Alexion's LAL Deficiency Drug Delayed 3 Months

James Radke, PhD

The Prescription Drug User Fee Act (PDUFA) date for Alexion’s Kanuma (sebelipase alfa), to treat lysosomal acid lipase (LAL) deficiency has been delayed to allow the FDA time to review new data they requested. The original PDUFA date of September 8, 2015 has been extended by 3 months (ie, December 8, 2015). 

The FDA asked the company for additional Chemistry, Manufacturing and Controls (CMC) information (that the company has provided). According to Alexion, the FDA extended the PDUFA date to allow additional time to review  the new CMC information. The FDA has not asked for additional clinical data.

Alexion acquired Kanuma following the purchase of Synageva earlier this year. In the video clip below, Don Wilson, MD, FNLA, of Cook Children's Physician Network in Fort Worth, Texas describes this rare condition and the need for an approved treatment. LAL deficiency is a rare, progressive disease that is due to patients having iineffective LAL.


Alexion Receives Notification of PDUFA Date Extension for Kanuma (sebelipase alfa) [news release]. Cheshire, CT: Alexion Pharmaceuticals inc; September 4, 2015.
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