Edgewise Therapeutics’ EDG-5506 on FDA fast track for Duchenne MD

Edgewise running multiple clinical trials of EDG-5506 for DMD, Becker MD

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

A person wearing a baseball cap speaks into a cone-shaped megaphone.

EDG-5506, an oral treatment being developed by Edgewise Therapeutics, has been granted fast track designation for Duchenne muscular dystrophy (DMD) by the U.S. Food and Drug Administration (FDA).

The agency gives this designation to experimental therapies that have the potential to fill unmet medical needs, with the aim of getting much-needed medications to market faster. With the designation, Edgewise will have access to perks like more frequent meetings with the FDA during the drug development process.

“Every day is important for individuals living with Duchenne and their families, and we are pleased that the FDA granted fast track designation,” said Kevin Koch, PhD, president and CEO of Edgewise, said in a company press release.

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EDG-5506 granted orphan drug, rare pediatric disease designations in 2023

Late last year, the FDA also granted EDG-5506 both orphan drug and rare pediatric disease designations, which are designed to incentivize companies to develop therapies for rare disorders like DMD and the other muscular dystrophies.

“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,” Koch said.

In DMD, muscle cells lack a protein called dystrophin, which normally acts like a shock absorber that cushions muscles against damage during movement. Without dystrophin, muscle cells accumulate more wear-and-tear damage over time, ultimately resulting in symptoms like muscle weakness and wasting.

Given orally, EDG-5506 is designed to block the activity of myosin, one of the key proteins involved in muscle contractions. The therapy is intended to slow DMD progression by making muscle movements more gentle so that muscles don’t accumulate as much wear and tear.

Edgewise is currently running multiple clinical trials of EDG-5506 in people with DMD. The Phase 2 LYNX study (NCT05540860) is testing multiple daily doses of EDG-5506 against a placebo in ambulatory boys, ages 4 to 9, with DMD, and recruitment is ongoing at sites in the U.S. Assessments will be made over two years.

In addition, a yearlong Phase 2 study called FOX (NCT06100887) will test two EDG-5506 doses against a placebo in DMD patients ages 6 to 14 who have previously received gene therapy. FOX will also run at sites across the U.S.

Edgewise is also developing EDG-5506 for Becker muscular dystrophy (BMD), a milder disorder where muscle cells have dystrophin protein, but the protein is either dysfunctional or at abnormally low levels. A trial in BMD called CANYON (NCT05291091) and its expansion to include 120 additional participants, GRAND CANYON, are ongoing with patients being recruited at sites in the U.S., the U.K., and the Netherlands.