BioCentury
ARTICLE | Clinical News

Cholbam cholic acid regulatory update

March 23, 2015 7:00 AM UTC

FDA approved an NDA for Cholbam cholic acid from Asklepion to treat bile acid synthesis disorders due to single enzyme defects in pediatric and adult patients and as an adjuvant to treat peroxisomal disorders, including Zellweger spectrum disorders. Retrophin Inc. (NASDAQ:RTRX, New York, N.Y.) said it will now purchase Cholbam from Asklepion. Under a January deal, Retrophin gained an exclusive right to purchase the cholic acid capsules following FDA approval. Retrophin expects the deal to close and be able to launch Cholbam in as few as 2-4 weeks. Retrophin declined to disclose Cholbam’s price.

Asklepion also received a Priority Review voucher for Cholbam under FDA’s pediatric disease voucher program, which will transfer to Retrophin. The program is intended to give pharma companies incentives to develop treatments for rare pediatric diseases. Retrophin said it has not yet decided how it will use the voucher. ...