Benzinga | Aug 25, 2020 08:46AM EDT

Sarepta Therapeutics Announces FDA Acceptance Of Casimersen New Drug Application For Patients With Duchenne Muscular Dystrophy Amenable To Skipping Exon 45

-- FDA grants Priority Review Status and sets regulatory action date for February 25, 2021 --

-- FDA has indicated it does not currently plan to hold an advisory committee meeting to discuss the application --

-- Received FDA's conditional approval of AMONDYS 45(tm) as brand name forcasimersen --

-- Casimersen has been studied for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne --

CAMBRIDGE, Mass., Aug. 25, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company's New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045) and provided a regulatory action date of February 25, 2021. The FDA has indicated it does not currently plan to hold an advisory committee to discuss the application. In addition, the Company has received conditional approval of AMONDYS 45 as the brand name for casimersen. Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is engineered to treat patients with Duchenne muscular dystrophy (DMD) who have genetic mutations that are amenable to skipping exon 45 of the dystrophin gene.

The Company submitted its NDA filing in June 2020 and requested priority review, which the FDA granted. The NDA included data from the casimersen arm of the ESSENCE study (also known as Study 4045-301), a global, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of casimersen in patients amenable to skipping exons 45. An interim analysis from ESSENCE demonstrated a statistically significant increase in dystrophin production as measured by western blot in patients who received casimersen compared to baseline and placebo. The study is ongoing and remains blinded to collect additional efficacy and safety data.

"The FDA's acceptance of our NDA for casimersen is an important step toward our goal of rapidly bringing therapies to patients living with Duchenne muscular dystrophy," said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. "If it is approved, casimersen, our third exon-skipping medicine in our PMO RNA-based platform, will offer treatment to the 8% of Duchenne patients who are amenable to exon 45 skipping."