Novartis, Genentech tout long-term data on muscular atrophy therapies
Andrzej Rostek
- Novartis (NYSE:NVS) and Roche (OTCQX:RHHBY) unit Genentech are touting long-term data for their respective spinal muscular atrophy (SMA) treatments.
- Novartis (NVS) said that data from a trial on its one-time gene therapy Zolgensma (onasemnogene abeparvovec) demonstrated that after presenting symptoms of SMA, children who received treatment maintained all previously achieved motor milestones for up to 7.5 years.
- Interim results from a second study that is to last 15 years that includes presymptomatic and symptomatic patients found all maintained motor milestones achieved during parent studies in the follow-up period.
- Four-year data on Genentech's Evrysdi (risdiplam) in patients between the ages of two and 25 years old with SMA found that motor function was sustained and the rate of adverse events continued to decrease over the period.
- The companies presented the data at the Muscular Dystrophy Association Clinical and Scientific Conference.
- Read why Seeking Alpha contributor LD Investments recently called Novartis (NVS) a hold.