Sarepta sold the priority review voucher for Exondys 51, a controversial treatment for Duchenne Muscular Dystrophy, to Gilead.
Sarepta, maker of the controversial Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), has sold its rare pediatric disease priority review voucher (PRV) to Gilead for $125 million. Sarepta announced the sale in a Feb. 21, 2017 press release, but did not disclose the buyer of the PRV. A Form 8-K from the Securities and Exchange Commission confirmed that Gilead would be purchasing the PRV from Sarepta. Gilead has not said for which drug it will use the voucher.
The voucher was acquired by Sarepta when Exondys 51 was approved by FDA for the treatment of DMD amendable to exon 51 skipping. Exondys 51 is an injectable treatment and is the first drug approved by FDA for DMD. The drug sparked controversy last year when FDA questioned its efficacy, citing small study size and insignificant increases in the production of dystrophin as potential reasons for concern.
Sarepta said the money from the sale of the PRV will be funneled back into the company, supporting their mission of treating boys with DMD. “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,” said Edward Kaye, Sarepta’s CEO, in a statement.
Source: Sarepta, Securities Exchange Commission