KER-065, an experimental Duchenne muscular dystrophy (DMD) drug developed by Keros Therapeutics, has been granted Orphan Drug designation from the U.S. Food and Drug Administration (FDA), according to a recently published press release.
About KER-065
KER-065 is an investigational drug that modulates enzymes involved in the regulation of muscle growth. Specifically, it decreases the activity of activin A and myostatin, two molecules that have a negative impact on the growth of skeletal muscles.
By inhibiting the biological effects of these enzymes, KER-065 is intended to promote muscle growth and increase strength and muscle regeneration, as well as reduce fat mass.
Learn more about DMD therapies
About the Orphan Drug designation
Orphan Drug designation is reserved for drugs intended to treat or prevent rare conditions that affect fewer than 200,000 people in the United States, or for diseases where there is no reasonable expectation that sales will recover research and development costs.
The aim of the designation is to provide incentives for pharmaceutical companies to develop treatments for rare diseases. Benefits include tax credits for qualified clinical research, federal grants to support clinical trials, seven years of market exclusivity upon approval and a waiver of FDA user fees. The FDA may also provide product-development support for designated drugs.
“Receiving Orphan Drug Designation for KER-065 highlights the significant unmet medical need for patients with DMD,” said Jasbir S. Seehra, president and chief executive officer of Keros Therapeutics. “This designation serves as a significant milestone for Keros as we advance KER-065 into a Phase 2 clinical trial in patients with DMD.”
Current DMD therapeutic landscape
Corticosteroids represent the current standard of care in DMD. There is substantial evidence that corticosteroids such as prednisone prolong ambulation and slow disease progression, but they are associated with important long-term adverse effects.
In recent years, more targeted, DMD-specific therapies, such as exon-skipping therapy and stop-codon readthrough therapy, have shown similar or greater efficacy than corticosteroids, with a more favorable safety profile.
Sign up here to get the latest news, perspectives, and information about DMD sent directly to your inbox. Registration is free and only takes a minute.
