- Illinois-based Marathon Pharmaceuticals on Tuesday said it had submitted an application to the FDA for approval of the off-patent steroid deflazacort for treatment of Duchenne muscular dystrophy (DMD).
- Deflazacort, a corticosteroid with anti-inflammatory properties, has been approved internationally for uses other than treating DMD and was previously studied as a possible treatment for the progressive muscle-wasting disease.
- Marathon presented data from a Phase 3 study of the drug at the annual meeting of the American Academy of Neurology in April, showing deflazacort appeared to improve muscle strength in DMD patients compared to a placebo at 12 weeks.
Hopes for a new treatment for the underlying causes of DMD have been repeatedly dashed over the past six months, as the FDA has rejected drugs from BioMarin Pharmaceutical and PTC Therapeutics.
An independent advisory committee for the FDA in April voted against recommending approval for another drug from Sarepta Therapeutics, eteplirsen. The FDA has not made a final decision on approval for the drug yet, blowing past its May 26 deadline. Earlier this month, the regulator asked Sarepta for more data on eteplirsen, raising hopes the drug may not be fully rejected.
Both eteplirsen and BioMarin's drisapersen were developed to boost the production of dystrophin, a crucial protein that helps muscle fibers function. Patients with DMD don't make enough dystrophin due to errors in the dystrophin gene.
Marathon believes deflazacort, on the other hand, can help boost muscle strength and performance on timed function tests through its steroidal properties. The FDA has granted the drug's application fast track status, along with orphan drug and rare pediatric disease designations.
In studies of the drug, the most common side effects were cushingoid appearance, weight gain, hirsutism, central obesity, and increased appetite.
“We recognize the difficulty the Duchenne community has had in obtaining deflazacort and look forward to working closely with the FDA as they review our application," said Jeff Aronin, CEO of Marathon.
The company says it currently provides deflazacort to DMD patients free of charge through an expanded access program.
New drug applications are typically reviewed for completeness by the FDA within 60 days.