Golden Ticket: United Therapeutics Grabs the Second Rare Pediatric Disease Priority Review Voucher With UNITUXIN Approval

March 10, 2015

By Kurt R. Karst –      

That’s right folks, another Golden Ticket has been awarded!  On March 10, 2015, FDA approved BLA 125516 for United Therapeutics Corporation’s (“UTC’s”) Unituxin (dinutuximab) in combination with granulocyte-macrophage colony-stimulating factor, interleukin-2 and 13-cis-retinoic acid, for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy.  With that approval, FDA granted UTC a Rare Pediatric Disease Priority Review Voucher (“Pediatric PRV”), which entitles the voucher holder to priority review (instead of a longer standard review) of a single NDA or BLA after the date of approval of the rare pediatric disease product application.  (A copy of UTC’s press release is available here.)

As we noted when FDA granted the first Pediatric PRV back in February 2014 – to BioMarin Pharmaceutical Inc. for VIMIZIM (elosulfase alfa) Injection – the Pediatric PRV was created by Section 908 of the 2012 FDA Safety and Innovation Act (“FDASIA”) (codified at FDC Act § 529), and is intended to encourage the development of treatments for rare pediatric diseases.  The program was inspired by the existing Tropical Disease PRV (“TD-PRV”) program set forth at FDC Act § 524, as added by the 2007 FDA Amendments Act of 2007; however, the TD-PRV differs from the Pediatric PRV program in several respects.  Last November, FDA issued draft guidance explaining the Agency’s implementation of the statutory Pediatric PRV provisions (see our previous post here). 

Significantly, the Pediatric PRV program has a sunset clause.  Under the statute (FDC Act § 52(b)(5)), no further vouchers can be awarded “after the last day of the one-year period that begins on the date” that the third voucher is granted.  So the next Golden Ticket granted by FDA will trigger the sunset provision.   But Congress may renew the Pediatric PRV program after this trial run as a part of the next omnibus FDA bill.    

For now, the big question is what will will UTC do with the Pediatric PRV.  PRVs are, after all, transferable and may be sold.  In July 2014, Sanofi and Regeneron paid BioMarin $67.5 million for its Pediatric PRV.  Late in 2014, Gilead Sciences, Inc. nearly doubled that figure when it paid Knight Therapeutics Inc. $125 million for its Tropical Disease PRV.  Another Tropical Disease PRV FDA granted with the 2012 approval of SIRTURO (bedaquiline) for tuberculosis has reportedly gone unused.