Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that the FDA has accepted its new drug application (“NDA”) seeking accelerated approval for its second Duchenne Muscular Dystrophy ("DMD") drug, golodirsen. The regulatory authority also granted priority review to the NDA with a decision expected by Aug 19, 2019.
The company had completed the submission of a rolling NDA in December last year. The candidate, golodirsen, also enjoys Orphan Drug designation for treating DMD.
Sarepta already has a marketed DMD drug, Exondys 51, which performed well since launch with sales set to reach $300 million in 2018. The approval of golodirsen will expand the patient population by 8%.
Sarepta’s shares have risen 4.1% in the past six months against the industry’s decline of 12.9% in the same time frame.
The NDA was submitted based on data from the phase I/II 4053-101 study, which demonstrated that treatment with golodirsen achieved 100% response rate with 10.7 times increase in mean dystrophin protein (muscle protein) from baseline. The increase in muscle protein was also better compared to three-times for Exondys 51.
Meanwhile, the ongoing phase III study, 4045-301 (ESSENCE) on golodirsen will serve as the post-marketing confirmatory study.
Sarepta has a strong pipeline of exon-skipping and gene therapy candidates, which are in various stages of development as a treatment for DMD. In fact, the company has about eight exon-skipping candidates in its pipeline, including golodirsen. Casimersen, the second-most advanced candidate, is being evaluated in a phase I/II Study 4045-101 for treating DMD patients aged 7 to 21 years. The company expects that successful development of its exon-skipping candidate will help it treat 75-80% of the DMD population.
The company is also developing gene therapies for DMD as well as central nervous system diseases (“CNS”). A phase I/II study is evaluating its micro-Dystrophin gene therapy, which has demonstrated potential as a treatment for patients with DMD. The company has also collaborated with partners to develop gene therapies.
In a separate press release, Sarepta and its partner, Lysogene, announced the initiation of a phase II/III study to evaluate gene therapy, LYS-SAF302, as a treatment for mucopolysaccharidosis Type IIIA, a progressive CNS disorder.
Currently, Sarepta is growing on the strong uptake of Exondys 51. However, competition in the DMD market is low as there are very few therapies available. Potential approval of golodirsen will also boost Sarepta’s prospects as it will expand the eligible patient population. However, competition is expected to intensify. Successful development of gene therapy candidates by Pfizer (PFE - Free Report) , Solid Biosciences (SLDB - Free Report) and Scholar Rock (SRRK - Free Report) may impact Sarepta’s top-line adversely.
Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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