EDG-5506 wins FDA orphan drug designation for DMD and BMD

Edgewise's oral therapy ultimately aims to slow disease progression over time

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

A man wearing a suit jacket and tie speaks at a podium with a microphone.

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to Edgewise Therapeutics‘ EDG-5506 as a potential treatment for Duchenne muscular dystrophy (DMD).

EDG-5506 also was awarded FDA orphan drug status for Becker muscular dystrophy (BMD) as a possible future therapy for that form of the genetic disease.

Both designations aim to speed the development of treatments for rare disorders, defined as those affecting fewer than 200,000 people in the U.S. Such status provides incentives, and in the case of orphan drug designation, the promise of market exclusivity, to the companies developing these therapies.

“Receiving orphan drug and rare pediatric disease designations are important milestones in advancing our novel small molecule therapeutic approach to treating individuals with Duchenne and Becker,” Kevin Koch, PhD, president and CEO of Edgewise, said in a company press release.

The experimental therapy ultimately aims to slow the progression of DMD and BMD over time.

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EDG-5506 helping to preserve muscles in Becker MD Phase 1 trial

FDA orphan drug designation awarded to therapy for rare diseases

The FDA’s orphan drug designation was created to incentivize companies to develop treatments for rare diseases where there might not be a strong economic incentive under unregulated free market conditions. The designation confers certain incentives to the therapy’s developer: Most notably, orphan drugs have a guarantee that, if they are ultimately approved, they will have seven years of market exclusivity without competition from other formulations or generics.

The rare pediatric disease designation similarly is awarded to therapies aiming to treat rare disorders — as its name implies, those that mainly affect children. Drugs given this designation are automatically given priority review, which means the FDA’s review of an application seeking approval is shortened by a few months.

If the therapy ultimately is approved, its developer also gets a voucher for priority review of another drug, which the company can either use itself or sell.

All three new statuses were welcomed by Edgewise.

“These regulatory designations highlight the urgent and critical need for new and better therapeutic options for people living with these rare, serious or life-threatening disorders,” Koch said.

EDG-5506 previously was granted FDA fast track designation for BMD, which is designed to hasten the development of potentially important new treatments.

These regulatory designations highlight the urgent and critical need for new and better therapeutic options for people living with these rare, serious or life-threatening disorders.

Both DMD and BMD are caused by mutations in the gene that provides instructions for making the protein dystrophin. BMD is characterized by an abnormal form of dystrophin, whereas in DMD, no dystrophin protein is made at all.

The dystrophin protein normally provides stability to muscle cells’ membranes during muscle contractions, helping to cushion the cells against wear-and-tear damage.

EDG-5506 is an oral therapy designed to make muscle contractions less forceful by blocking the activity of myosin, one of the main proteins that drives muscle contractions. The theory is that making muscle contractions less forceful may lead to less wear-and-tear damage, ultimately slowing disease progression over time.

Data from a Phase 1 study called ARCH (NCT05160415) indicated that most men with BMD had stable or improved motor function after a year of treatment with EDG-5506. That’s a notable departure from the typical course of the disease, in which motor function gets steadily worse as time goes on.

Edgewise now is running a Phase 2 trial called CANYON (NCT05291091) that is testing EDG-5506 against a placebo in people with BMD ages 12 to 50. The company recently announced a pivotal expansion to the trial, dubbed GRAND CANYON, with plans to enroll an additional 120 adults with BMD; recruitment is ongoing at sites in the U.S., the U.K., and the Netherlands.