Rare Disease Report

Priority Review Voucher Price Tag is Now $245 Million

MAY 26, 2015
James Radke, PhD

The price of priority review vouchers continues to rise. Today, Retrophin announced it has sold its Rare Pediatric Disease Priority Review Voucher to Sanofi for $245 million. 

That is much higher than the $67.5 million and $125 million that Biomarin and Knight Therapeutics received for their vouchers last year from Regeneron and Gilead, respectively.

The priority review voucher was given to Retrophin following the approval of Cholbam (cholic acid) was approved by the US Food and Drug Administration (FDA) on March 17, 2015 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 Priority Review Voucher program was introduced to encourage companies to develop drugs for rare pediatric conditions or neglected tropical diseases. If the company’s drug gets approved for a pediatric or neglected tropical disease, the FDA can give them a voucher that they can then use for future drug applications. A priority review designation by the FDA means the drug will be reviewed in 6 months instead of the standard 10 months. And the vouchers can be applied to ANY future drug application by the company. Normally, a priority review designation would only be given to a drug that is for a 'serious conditon' and has demonstrated 'the potential to be a significant improveint in safety and effectiveness'.

The Priority Review Voucher is also transferable and that is what Retrophin decided to do with their voucher—sell it. And Sanofi believes that getting one of their drugs to market 4 months earlier is worth $245 million.

Upon closing of the transaction, Retrophin will receive a payment of $150 million, followed by two equal payments of $47.5 million in 2016 and 2017.


Retrophin Agrees to Sell Priority Review Voucher to Sanofi [press release]. San Diego, CA: Retrophin, Inc; May 27, 2015. http://www.businesswire.com/news/home/20150527005772/en/Retrophin-Agrees-Sell-Priority-Review-Voucher-Sanofi#.VWW7Oqb1h9I

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