The Food and Drug Administration (FDA) has granted Fast Track designation to EDG-5506 for the treatment of patients with Becker muscular dystrophy.
Becker muscular dystrophy is a rare, genetic neuromuscular disorder that causes progressive muscle weakness and damage and primarily affects the skeletal and cardiac muscles. The disorder is caused by a mutation of the X-linked dystrophin gene.
EDG-5506 is an oral small molecule myosin inhibitor designed to protect and improve the function of dystrophic skeletal muscle fibers by selectively limiting injurious hypercontraction stress caused by the absence of functional dystrophin.
The designation was supported by preclinical data and an ongoing phase 1 trial (ClinicalTrials.gov Identifier: NCT04585464) evaluating the safety, tolerability and pharmacokinetics of EDG-5506 in adult healthy volunteers and adults with BMD. The Company expects to have phase 1 trial data later this year.
“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,” said Kevin Koch, PhD, President and CEO of Edgewise. “We look forward to working closely with the FDA towards establishing EDG-5506 as the potential first muscle-directed therapy for individuals with Becker.”
The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to therapies that are expected to have an impact on factors such as survival and daily functioning.
Edgewise receives U.S. FDA Fast Track designation for EDG-5506 for the treatment of individuals with Becker muscular dystrophy (BMD). News release. Edgewise Therapeutics, Inc. Accessed August 17, 2021. https://investors.edgewisetx.com/news/news-details/2021/Edgewise-Receives-U.S.-FDA-Fast-Track-Designation-for-EDG-5506-for-the-Treatment-of-Individuals-with-Becker-Muscular-Dystrophy-BMD/default.aspx.