Sarepta Therapeutics, a commercial-stage developer of innovative RNA-targeted therapeutics, has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV). Sarepta received the PRV when EXONDYS 51TM was approved by the FDA for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping.
The voucher was awarded by the FDA under a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. With the passage of the 21st Century Cures Act, this PRV program has been extended through September 30, 2020.
As part of the agreement, Sarepta will receive an upfront payment of $125 million upon the closing of the transaction, which is subject to customary closing conditions and is expected to occur following expiration of the applicable U.S. antitrust clearance requirements.
“Our mission at Sarepta Therapeutics is to treat more boys with Duchenne muscular dystrophy,” said Edward Kaye, Sarepta’s chief executive officer. “The sale of the PRV provides an important source of non-dilutive capital to support the rapid advancement of our follow on exon skipping candidates and next generation RNA targeted antisense platform.”
The Rare Pediatric Disease Priority Review Voucher program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases. A PRV may be issued to the sponsor of a rare pediatric disease product application and would entitle the holder to priority review of a single New Drug Application or Biologics License Application, which reduces the target review time and could lead to an expedited approval. The sponsor receives the PRV upon approval of the rare pediatric disease product application and it can be sold without limitation, subject to applicable FDA requirements for filing and use.