FDA Approves Oral Treatment for Spinal Muscular Atrophy
Retrieved on:
Friday, August 7, 2020
This is the second drug and the first oral drug approved to treat this disease.
Key Points:
- This is the second drug and the first oral drug approved to treat this disease.
- SMA is a hereditary disease that causes weakness and muscle wasting because patients lose lower motor neurons (nerve cells) that control movement.
- The efficacy of Evrysdi for the treatment of patients with infantile-onset and later-onset SMA was evaluated in two clinical studies.
- After 23 or more months of treatment, 81% of patients were alive without permanent ventilation, which is a noticeable improvement from typical disease progression without treatment.