The US Food and Drug Administration (FDA) has granted fast track designation to Edgewise Therapeutics’ investigational drug, EDG-5506, for Duchenne muscular dystrophy.
The designation expedites the development and review of EDG-5506, an oral small molecule for the prevention of contraction-induced muscle damage in dystrophinopathies such as Duchenne and Becker muscular dystrophy.
The FDA had previously recognised the potential of the therapy by granting it orphan drug and rare paediatric disease designations for Duchenne. It had also received fast track status for the treatment of Becker muscular dystrophy.
EDG-5506 is being evaluated in ongoing placebo-controlled clinical trials including the Phase II LYNX trial in children with Duchenne aged four to nine years and the Phase II FOX study in children and adolescents who previously received gene therapy.
The three-month controlled, dose-ranging findings from the LYNX trial are anticipated in the second quarter of 2024 and will inform the Phase III dose.
LYNX is analysing the safety, pharmacokinetics and biomarkers of muscle damage.
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In addition to the LYNX and FOX trials, the company is completing subject enrolment for the CANYON cohort of its Phase II trial in adult males with Becker muscular dystrophy.
This cohort will assess the safety and impact on function and muscle damage biomarkers in adult males with Becker.
The GRAND CANYON cohort, an extension of this trial, is enrolling an additional 120 adult participants.
Edgewise president and CEO Kevin Koch stated: “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.”
