Stakeholders aiming to renew pediatric voucher program

FDA issued its third Priority Review voucher under its pediatric disease voucher program Tuesday, which triggers a 12-month clock until the pilot program ends. The program is intended to give pharma companies incentives to develop treatments for rare pediatric diseases.

The voucher went to Asklepion Pharmaceuticals LLC (Baltimore, Md.), which received FDA approval for Cholbam cholic acid to treat pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders including Zellweger spectrum disorders. Retrophin Inc. (NASDAQ:RTRX) acquired the voucher and the drug from Asklepion. ...