Roche Announces New Positive Data on the Long-Term Efficacy and Safety of Evrysdi in SMA


(RTTNews) – Roche Holding AG (RHHBY) on Wednesday announced new positive long-term efficacy and safety data for Evrysdi (risdiplam) in a large population of people with spinal muscular atrophy or SMA.

The company noted that long-term efficacy data from the pivotal SUNFISH study confirms that increases in motor function are maintained at three years, while adverse effects decline over the same period.

Evrysdi is a survival motor neuron 2 or SMN2 splicing modifier designed to treat SMA caused by mutations in chromosome 5q that result in SMN protein deficiency. Evrysdi is taken daily at home in liquid form by mouth or by feeding tube.

Presentations included new three-year data from the SUNFISH study that confirmed the long-term efficacy and safety of Evrysdi in a large population of people aged 2 to 25 years with type 2 SMA or type 3. Additional presentations included two-year exploratory efficacy data from SUNFISH Part 2, demonstrating improvement or stabilization of motor function with Evrysdi compared to an untreated external control group.

Evrysdi was well tolerated over the three-year period of the SUNFISH study. The overall rate of adverse events in SUNFISH decreased over three years, and a downward trend in the rate of serious adverse events (was observed in the third year of treatment.

Roche also announced updated interim data from the RAINBOWFISH study in pre-symptomatic babies with SMA aged less than two months. The results demonstrate that the majority of babies treated with Evrysdi for at least 12 months were able to stand and walk within the time frames typical of healthy babies.

The data was presented at the Muscular Dystrophy Association or MDA Clinical and Scientific Conference, March 13-16, 2022.

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