New Life for the Pediatric Priority Review Voucher Program

By Alexander J. Varond –

On December 18, Congress extended the rare pediatric disease priority review voucher (pediatric PRV) program out to September 30, 2016. The extension was granted as part of Congress’s budget deal. Although modest, it has been welcomed by industry. Many hope, however, that the six-month extension is merely a stopgap for a more robust extension of the program.

Since March 2015, there has been much speculation regarding if and when the pediatric PRV program would be renewed. FDA’s pediatric PRV program’s 1-year sunset provision was triggered when FDA issued the third PRV for Cholbam (see our previous post here). A number of attempts have been made to renew the program ahead of the March 2016 sunset: Representative Butterfield proposed extending the program in the Advancing Hope Act, the Senate proposed a corresponding but different bill, and the House of Representative’s 21st Century Cures Act also would breathe new life into the program. We blogged about these efforts here, here, and here.

Sanofi Redeems its Second Pediatric PRV

Sanofi recently redeemed its second PRV when it submitted an NDA for its fixed-ratio combination of insulin glargine and lixisenatide to treat Type II diabetes. Sanofi purchased the pediatric PRV from Retrophin Inc. in May for $245 million. This is the second pediatric PRV the company has redeemed. Sanofi appears to have used the PRV in an attempt to beat Novo Nordisk’s once-daily single-injection combination of Tresiba (insulin degludec) and Victoza (liraglutide), Xultophy, to market. Novo Nordisk submitted its NDA for Xultophy in September 2015. Earlier this year, Sanofi successfully used a pediatric PRV for its PCSK9 inhibitor, Praluent, beating Amgen’s PCSK9, Repatha, to market.