Keros Therapeutics Secures Orphan Drug Designation for DMD Therapy
Keros Therapeutics just earned a major regulatory win: the U.S. FDA has granted Orphan Drug designation to its investigational therapy KER-065, designed to treat Duchenne muscular dystrophy (DMD).
What Is KER-065?
KER-065 is a ligand trap—a novel therapeutic approach that blocks myostatin and activin A, two proteins linked to muscle breakdown.
The goal:
- Stimulate muscle regeneration
- Improve strength and bone health
- Reduce fibrosis
- Lower excess body fat
The therapy is now advancing into a Phase 2 clinical trial for DMD patients.
The Urgent Need
- DMD prevalence: Roughly 1 in 3,500 boys worldwide.
- Cause: Mutations in the dystrophin gene → loss of muscle stability.
- Impact: Progressive muscle degeneration, loss of mobility, breathing issues, and eventually heart failure.
- Current challenge: Limited treatment options with only partial impact on slowing disease progression.
Why Orphan Drug Status Matters
This designation signals two things:
- Recognition of unmet medical need for DMD therapies.
- Practical incentives for Keros, including:
- Tax credits for clinical trials
- Waiver or reduction of FDA fees
- Seven years of market exclusivity if approved
Looking Ahead
For Keros, this milestone is more than regulatory—it’s momentum. With Phase 2 trials on the horizon, KER-065 could emerge as a new therapeutic option for one of the toughest genetic muscle diseases.
For patients and families, it represents something more powerful: hope backed by science.
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