European agency grants orphan status to Edgewise drug

BOULDER — The European Medicines Agency has granted orphan drug designations to one of Edgewise Therapeutics Inc.’s, (Nasdaq: EWTX), muscular dystrophy drugs.

The company’s drug, sevasemten, is used in the treatment of Becker muscular dystrophy and Duchenne muscular dystrophy. Sevasemten is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage, the company said in a press statement. Sevasemten is in late-stage clinical trials for individuals with Becker and is also being studied in Duchenne.

“Receiving orphan drug designation in the U.S. and now in Europe highlights alignment across regions on the urgent and critical need for new and better therapeutic options for people living with Becker or Duchenne,” Kevin Koch, president and CEO of Edgewise, said in a written statement. “We are focused on advancing these muscle-targeted therapeutics as quickly as possible through clinical development for these communities.”

The European designation grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect no more than five in 10,000 people in the European Union, among other criteria. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, access to centralized authorization procedure and up to 10 years of market exclusivity in the European Union, if approved.

Sevasemten has previously achieved notable milestones by securing U.S. Food and Drug Administration orphan drug designation for the treatment of Becker and Duchenne, rare pediatric disease designation for the treatment of Duchenne, and fast track designations for the treatment of Becker and Duchenne.

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