The US Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for the treatment of individuals with becker muscular dystrophy (BMD), Edgewise Therapeutics announces.

EDG-5506, a selective, small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers in DMD and BMD, is advancing in a Phase 1 clinical trial, according to the company in a media release.

“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. 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.

“We look forward to working closely with the FDA towards establishing EDG-5506 as the potential first muscle-directed therapy for individuals with Becker.”

— Kevin Koch, PhD, president and chief executive officer, Edgewise Therapeutics

“I am pleased to see that the FDA recognizes Becker muscular dystrophy as a serious disease and that the team at Edgewise is acknowledging the important unmet need in Becker.”

— Craig McDonald, MD, Professor and Chair, Department of Physical Medicine & Rehabilitation, University of California Davis

Fast Track Program

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 robust preclinical data package and an ongoing Phase 1 clinical trial (NCT04585464). The Company expects to have Phase 1 topline Multiple Ascending Dose (MAD) data in healthy volunteers and in individuals with BMD later in 2021, the release continues.

About Becker Muscular Dystrophy

BMD is a serious, progressively debilitating, and potentially fatal inherited neuromuscular 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.

BMD typically presents with juvenile onset of muscle wasting and progressive symmetrical, proximal muscle weakness, calf hypertrophy, activity-induced muscle cramping and elevated creatine kinase (CK) activity. While the course of BMD is variable, it is unidirectional in terms of the inevitable progressive limb weakness resulting in severe disability. BMD is also associated with early mortality from cardiac disease that is disproportionately severe compared to that seen in DMD.

The incidence of BMD is approximately 1 in every 18,450 live male births. It is estimated that there are between 4,000–5,000 individuals with BMD in the US, with similar numbers of individuals living with BMD in Europe. 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 US.

[Source(s): Edgewise Therapeutics, Business Wire]