- Roche Holding AG's (OTC:RHHBY) Genentech has released new long-term data on its oral drug for spinal muscular atrophy (SMA).
- After three years of treatment, 91% of children who began taking the drug at ages 1-7 months were alive (n=58).
- In addition, of 48 children evaluated, 32 maintained, and four gained the ability to sit without support.
- No infant who gained the ability to sit without support lost this ability after three years of treatment. The majority of infants treated with Evrysdi maintained the ability to feed orally and swallow up to month 36.
- Also Read: Roche's Evrysdi Shows Long-Term Efficacy, Safety In Broader Spinal Muscular Atrophy Population.
- Infants with SMA have a genetic mutation to their SMN1 gene, which encodes a protein crucial to muscle movement. Children with SMA may struggle with sitting up, swallowing, or breathing.
- Evrysdi works by promoting the other SMN gene, SMN2, to make more of that essential protein.
- The company noted no treatment-related adverse events from the long-term FIREFISH study. The most common adverse events were fever, upper respiratory tract infection, and pneumonia.
- The rate of severe adverse events decreased over time.
- Price Action: RHHBY shares are down 0.58% at $46.31 during the market session on the last check Friday.
