Sarepta Therapeutics said today it will sell its Rare Pediatric Disease Priority Review Voucher (PRV) to Gilead Sciences for $125 million upfront.
Sarepta obtained the voucher when the FDA in September approved the company’s Exondys 51™ (eteplirsen) as a treatment for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
Gilead’s identity as the buyer was disclosed by Sarepta in a regulatory filing. Gilead emerged from “multiple pharmaceutical and biotech companies” to which Sarepta said it reached out during an “extensive sales process” overseen by Credit Suisse.
“The sale of the PRV provides an important source of nondilutive capital to support the rapid advancement of our follow-on exon-skipping candidates and next-generation RNA-targeted antisense platform,” Sarepta CEO Edward Kaye, M.D., said in a statement.
Sarepta’s pipeline includes potential treatments for DMD amenable to exon 53 skipping (the Phase II candidate SRP-4053) and amenable to exon 45 skipping (Phase I candidate SRP-4045)—as well as discovery and preclinical drug candidates designed to skip exons 44, 52, 50, 43, 55, 8, and 35.
Less than a month after Exondys 51 was approved, Sarepta expanded its pipeline by acquiring rights to the pipeline of Summit Therapeutics, led by Phase II DMD candidate ezutromid, for Europe, Turkey, and the Commonwealth of Independent States. That deal could generate up to $562 million-plus for Summit.
The FDA approved Exondys 51 despite recommendations by two advisory committees against approving the treatment and over objections from some administrators. Those administrators included the then-director of the FDA’s Office of New Drugs (OND), John K. Jenkins, M.D., subsequently retired from the federal government.
The FDA awarded the voucher through a program designed to promote development of new drugs and biologics for preventing and treating rare pediatric diseases. The program has been extended through September 30, 2020, as a result of the 21st Century Cures Act, enacted last year.
Sarepta said its sale of the voucher was subject to customary closing conditions and is expected to occur following expiration of U.S. antitrust clearance requirements.