A US Food and Drug Administration’s (FDA) panel of experts has recommended Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) investigational gene therapy for accelerated approval.

The Cellular, Tissue and Gene Therapies advisory committee voted eight to six in support of SRP-9001 (delandistrogene moxeparvovec) as a treatment for ambulatory DMD patients with a confirmed mutation in the DMD gene.

DMD is a rare and inherited X-chromosome-linked disease that results in the lack of dystrophin protein, which is required to strengthen and protect muscles.

Over time, this causes progressive loss of muscle strength, with most patients requiring full-time use of a wheelchair by their early teens. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure.

The disease almost exclusively affects males, occurring in approximately one in every 3,500-5,000 newborn males worldwide.

SRP-9001 is designed to address the underlying cause of DMD through the targeted production of functional components of dystrophin in muscle tissue.

The committee’s positive vote is “based on the evaluation of the totality of evidence including the SRP-9001 product design as well as biological and empirical data,” Sarepta said.

The FDA will now consider the panel’s advice as it makes a decision regarding the potential approval of SRP-9001, with a decision expected later this month.

Doug Ingram, Sarepta's president and chief executive officer, said: “Today’s advisory committee outcome is extremely important to the patient community, who are in urgent need of new therapies.

“With the 29 May action date our top priority, we will work collaboratively with the FDA to complete the review of our Biologics License Application for SRP 9001."

In November last year, Sarepta and partner Hansa Biopharma AB announced plans to initiate a clinical study to assess a pre-treatment to SRP-9001 in patients with pre-existing IgG antibodies, which can be a barrier to treatment.

Imlifidase has already demonstrated an ability to reduce pre-existing IgG antibodies to rAAVrh74 in preclinical work conducted by the two companies, allowing for safe and successful administration of SRP-9001.

Reference：
https://www.pmlive.com/pharma_news/fda_advisory_committee_backs_sareptas_duchenne_muscular_dystrophy_gene_therapy_1491643