FDA Grants Edgewise Fast Track Designation for Becker Muscular Dystrophy Therapy

The U.S. Food and Drug Administration granted Edgewise Therapeutics Fast Track designation for its experimental therapy EDG-5506 for the treatment of individuals with the rare neuromuscular disease Becker muscular dystrophy.

Photo: Kevin Koch, president and CEO of Edgewise

Becker muscular dystrophy (BMD) is a serious, progressively debilitating, and potentially fatal inherited disorder. BMD results from truncation or mutation of the X-linked dystrophin gene yielding unstable and/or dysfunctional dystrophin expression in muscles. Individuals with BMD, typically males, have ongoing muscle fiber (myofiber) degeneration that eventually leads to fibrosis, progressive loss of skeletal muscle function, and that can lead to severe disability and early death. Despite the seriousness of the disease, for many with BMD, the disease remains one of considerable unmet medical need as there are no approved therapies in the U.S.

EDG-5506 is an orally administered small molecule designed to address the root cause of dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action to selectively limit injurious hypercontraction stress caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is expected to be used as a single agent therapy, but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development.

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill an unmet medical need. The Fast Track Program supports important new therapeutics to reach patients earlier. The designation is granted to therapeutics that offer potential to meaningfully impact survival, day-to-day functioning, or if left untreated, progression of the condition. Therapeutics that receive this designation receive a number of benefits that include more frequent meetings with the FDA to discuss development of the drug candidate and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.

The decision to give EDG-5506 Fast Track designation was supported by a preclinical data package and an ongoing phase 1 clinical trial. The company expects to have phase 1 topline multiple ascending dose data in healthy volunteers and in individuals with BMD later in 2021.

“The FDA’s decision to grant EDG-5506 Fast Track designation underscores the urgency to address a significant unmet medical need for individuals with Becker muscular dystrophy,” said Kevin Koch, president and CEO of Edgewise. “Becker is a serious, progressive disease that leads to severe disability, including loss of ambulation and heart disease that has substantial impact on day-to-day function in many individuals.”

Author: Rare Daily Staff