FDA Approves Retrophin's Cholbam For Rare Bile Acid Synthesis Disorders

The U.S. Food and Drug Administration approved Retrophin Inc.'s Cholbam for the treatment of rare bile acid synthesis disorders caused by single enzyme defects, the company announced on Wednesday. As a result of the approval, Retrophin will exercise its right to acquire from Asklepion Pharmaceuticals LLC all worldwide rights to the treatment for a one-time cash payment of $28 million and 661,278 Retrophin shares. The FDA has also granted Asklepion an expedited review process for any new drug application in the form of a Rare Pediatric Disease Priority Review Voucher, or Pediatric PRV, a special provision used to encourage the development of treatments for rare pediatric diseases. The Pediatric PRV will be transferred to Retrophin as part of the deal. Cholbam will have seven years exclusivity in the U.S. Retrophin shares were halted in premarket trade, but have gained 20.6% in the year so far, while the S&P 500 has gained 0.8%.