Roche announced new long-term data for Evrysdi from the open-label extension of the pivotal FIREFISH study, “reinforcing its sustained efficacy and safety profile” in children with Type 1 spinal muscular atrophy, or SMA. FIREFISH is a two-part study in babies aged 1-7 months at the time of enrolment. After four years of treatment with Evrysdi, many of the babies, now young children, continued to improve their ability to sit, stand and walk without support. All the Evrysdi-treated children who were alive at the time of the primary analysis were still alive at month 48. Additionally, the majority of infants maintained their ability to feed by mouth and swallow up to month 48, Roche stated.
Elevate Your Investing Strategy:
- Take advantage of TipRanks Premium at 55% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence.
Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
See Insiders’ Hot Stocks on TipRanks >>
Read More on RHHBY:
- Four-year follow up data for Roche’s Evrysdi show continued increase in number of children with a severe form of spinal muscular atrophy (SMA) able to sit, stand and walk
- Genprex receives FDA Fast Track Designation for REQORSA/Tecentriq combo
- Regeneron price target lowered to $789 from $816 at RBC Capital
- Regeneron Stock (NASDAQ:REGN) Sinks; FDA Snubs Higher-Dose Eye Drug
- Roche turns positive after Regeneron issued CRL for aflibercept 8 mg BLA