The FDA has also granted Rare Pediatric Disease Designation for the treatment of Duchenne, Fast Track designation for the treatment of Becker, and Orphan Drug Designation to EDG-5506
US FDA grants fast track status to Edgewise's Duchenne therapy. (Credit: Towfiqu barbhuiya on Unsplash)
Edgewise Therapeutics has been granted Fast Track designation by the US Food and Drug Administration (FDA) for EDG-5506 to treat Duchenne muscular dystrophy.
EDG-5506 is an investigational orally administered small molecule intended to inhibit contraction-induced muscle damage in dystrophinopathies, including Duchenne and Becker muscular dystrophy (Becker).
Before this, the FDA granted Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne, Fast Track designation for the treatment of Becker, and Orphan Drug Designation (ODD) to EDG-5506.
The latest designation will facilitate the development and expedite the review of EDG-5506.
It will also get more frequent meetings with the health regulator to discuss the development of the drug candidate and a rolling review of the marketing application for securing FDA approval.
The ongoing Duchenne trials include the Phase 2 placebo-controlled LYNX trial, which is open to children with the disease aged four to nine years, and the Phase 2 placebo-controlled FOX trial, which is open to children and adolescents with the disease who have received gene therapy in the past.
Once the Phase 3 dose is determined, the company plans to disclose LYNX 3-month controlled dose-ranging data in the second quarter of 2024.
Edgewise president and CEO Kevin Koch said: “Every day is important for individuals living with Duchenne and their families, and we are pleased that the FDA granted Fast Track designation.
“Our roster of regulatory designations granted to EDG-5506 highlights the urgent and critical need for new and better therapeutic options for people living with these rare and life-threatening muscle disorders.”
A Phase 2 trial, cohort named CANYON, which is currently enrolling participants, has been completed.
The trial evaluated the safety of EDG-5506 and its effects on function and biomarkers of muscle damage in adult males with Becker.
The study has been expanded to include an additional 120 adult participants in a cohort named GRAND CANYON.