- Retrophin is selling a priority review voucher, which it purchased from Askepion, to Sanofi for $150 million upfront in addition to two $47.5 million installments.
- The Rare Pediatric Disease Priority Review Voucher Program was enacted in 2012 to encourage development of drugs for pediatric diseases, which affect 200,000 children or fewer in the U.S. To date, the FDA has issued six priority review vouchers, including three pediatric vouchers.
- The benefit of receiving such a voucher is that the standard 10-month review process is expedited to six months.
Given the fact that there are only three Rare Pediatric Disease Priority Review Vouchers in existence, they have been rising in value. Until now, the biggest sale was $125 million. Sanofi now has the ability to shorten its FDA review timeframe by four months.
According to Retrophin, the company received the Pediatric PRV when Cholbam was approved by the U.S. Food and Drug Administration (FDA) for the treatment of pediatric and adult patients suffering from bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders).
The stock is up almost 12% on the news.