Sarepta's Elevidys expansion bid nabs FDA priority review, with no adcomm planned

Ever since Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy Elevidys scored its initial approval in a restricted patient population, the company has aimed to expand the drug's reach.

Despite a primary endpoint miss in a pivotal study, it looks like Sarepta’s dream is one step closer to coming true as the FDA accepted the company's efficacy supplement under a priority review. The agency plans to make a decision on Sarepta's label expansion filing by June 21.

In addition, the agency confirmed that it won't convene an advisory committee meeting to discuss the potential new label.

The company’s efficacy supplement aims to expand the therapy’s current label, which covers ambulatory patients ages 4 to 5, to the broader population of “DMD patients with a confirmed mutation in the DMD gene,” the company said in its Friday press release.

Additionally, the new filing hopes to convert the current accelerated approval to a traditional nod.

“Understanding that every day matters to families living with Duchenne, we will work with our regulatory counterparts to successfully complete this review as rapidly as possible," Sarepta CEO Doug Ingram said in the release.

The new goal date is almost exactly a year after Elevidys first crossed the FDA finish line in its restricted population. That decision followed several delays and an advisory committee on the company's original filing.

Before approval, the FDA raised concerns that the “clinical studies conducted to date do not provide unambiguous evidence” that the treatment is “likely beneficial” for patients included in the drug's current label. Nonetheless, the FDA followed the advisory committee’s narrow recommendation in favor of the therapy’s risk-benefit profile.

More recently, the company’s phase 3 study in support of the potential expansion missed its primary endpoint, which was a measure of motor function called the North Star Ambulatory Assessment. Still, Ingram maintained that the study “met the standard” to show “evidence of effectiveness,” the CEO said in October.  

Despite the label restriction, the $3.2 million gene therapy generated $69.1 million during its first quarter on the market, more than exceeding analysts’ expectations of between $20 million and $24 million.

On Sarepta’s third-quarter earnings call, Ingram pointed out that parents of patients outside of the currently approved age range are “probably even more compelled” to want to start the therapy and are awaiting their chance upon the potential label expansion.

Sarepta shares jumped 10.75% on Friday morning with the announcement.