New data for Evrysdi demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophy. Genentech/Roche

Additional presentations included exploratory two-year efficacy data from SUNFISH Part 2, demonstrating improvement in, or stabilization of, motor function with Evrysdi compared to an untreated external control group.

Genentech also announced updated interim data from the RAINBOWFISH study in pre-symptomatic babies with SMA under two months of age. The data were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 13-16, 2022.

“The positive long-term efficacy and safety results for Evrysdi in this broad SMA population are important for physicians as they consider Evrysdi as a treatment option for their patients,” said Laurent Servais, M.D., Ph.D., Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre. “In treating people with SMA, our aim is to enable or preserve their independence and patients in the SUNFISH study reported continuous improvement or stabilization in the level of help needed for daily living.”

In the SUNFISH study, the increase in Motor Function Measure 32 (MFM-32) total score from baseline previously observed at year one was maintained through year three in people treated with Evrysdi. The increases in Revised Upper Limb Module (RULM) and Hammersmith Functional Motor Scale Expanded (HFMSE) total scores from baseline were also sustained between year one and year three. Evrysdi was well-tolerated over the three-year time period in the SUNFISH study. The overall rate of adverse events (AEs) in SUNFISH decreased over three years, and a trend towards a lower rate of serious adverse events (SAEs) was observed in the third year of treatment. Overall, AEs and SAEs were reflective of the underlying disease and no treatment-related AEs led to withdrawal from the study.

In addition, for the first time, an external comparator analysis has been done for SUNFISH two-year data with an untreated control group. The weighted exploratory analyses of MFM total scores showed that in SUNFISH Part 2, the proportion of patients demonstrating a marked improvement (change greater than 3 points) or stabilization (change greater than 0 points) were more likely in patients who were on Evrysdi for 24 months than those in the untreated comparator group (p=0.025 and p=0.002 respectively).

Updated interim data from the RAINBOWFISH study were also shared, demonstrating the safety and efficacy of Evrysdi for newborns. In January, the FDA granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi to treat pre-symptomatic babies under two months of age with SMA. To date, more than 5,000 people have been treated with Evrysdi in clinical trials, compassionate use or real-world settings. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.