Retrophin acquires Cholbam (cholic acid) from Asklepion Pharmaceuticals following FDA approval for treatment for bile acid disorders.

Retrophin, Inc.has announced that the FDA has approved Cholbam (cholic acid) capsules, the first FDA approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders). As a result of the approval, Retrophin will exercise its right to purchase from Asklepion Pharmaceuticals, LLC all worldwide rights, titles, and ownership of Cholbam and related assets. Retrophin will pay Asklepion a one-time cash payment of $27 million, in addition to approximately 661,278 shares of Retrophin common stock (initially valued at $9 million at the time of the agreement), which assumes Cholbam received an approval for a cerebrotendinous xanthomatosis (CTX) indication. Asklepion will also be eligible to receive up to $37 million in cumulative sales milestones, as well as tiered royalties based on future net sales of Cholbam.

The FDA also granted Asklepion a Rare Pediatric Disease Priority Review Voucher ("Pediatric PRV"), a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. This voucher is designed to be transferable or sold and provides the bearer with an expedited FDA review for any new drug application. The Pediatric PRV will be transferred to Retrophin under the original terms of the agreement with Asklepion.

Comment: Cholbam complements Retrophin's existing bile acid therapy, Chenodal (chenodeoxycholic acid), and will position that company as the leading provider of treatments for patients with bile acid synthesis and peroxisomal disorders. Cholbam will have seven years market exclusivity in the United States conferred by its designation as an orphan drug.