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

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Evrysdi (risdiplam) has received additional data from Roche in the treatment of spinal muscular atrophy (SMA). New three-year data from the SUNFISH research were presented, confirming Evrysdi’s long-term efficacy and safety in a broad sample of adults aged 2 to 25 with Type 2 or Type 3 SMA. Exploratory two-year efficacy findings from SUNFISH Part 2 were also presented, suggesting that Evrysdi improved or stabilized motor function when compared to an untreated external control group. Roche also released updated intermediate results from the RAINBOWFISH research, which looked at pre-symptomatic newborns with SMA who were under the age of two months. The findings were presented at the Muscular Dystrophy Association’s (MDA) Clinical and Scientific Conference, which will take place from March 13 to 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. In treating people with SMA, our aim is to enable or preserve their independence and patients in the SUNFISH study reported continuous improvement or stabilisation in the level of help needed for daily living,”

said Laurent Servais, M.D., Ph.D., Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre. 

The rise in Motor Function Measure 32 (MFM32) total score from baseline reported at year one in patients treated with Evrysdi was maintained through year three in the SUNFISH trial. Between year one and year three, the increases in total scores on the Revised Upper Limb Module (RULM) and Hammersmith Functional Motor Scale Expanded (HFMSE) from baseline were likewise maintained.

The SUNFISH trial found that Evrysdi was well tolerated over a three-year period. Over the course of three years, the overall rate of adverse events (AEs) in SUNFISH reduced, with a tendency toward a lower rate of serious adverse events (SAEs) in the third year of treatment. Overall, AEs and SAEs were consistent with the underlying condition, and no treatment-related AEs resulted in study withdrawal.

In addition, an external comparator analysis of SUNFISH two-year data with an untreated control group was performed for the first time.

In SUNFISH Part 2, weighted exploratory analyses of MFM total scores revealed that patients on Evrysdi for 24 months were more likely than those in the untreated comparison group to have a considerable improvement (change of 3 points) or stabilization (change of 0 points). (p=0.002 and 0.025, respectively)

“We are pleased that these long-term results further reinforce the safety and efficacy of Evrysdi and it is especially encouraging to see that adverse events decreased over time. We remain committed to working towards continued access to Evrysdi for all appropriate patients with this progressive disease.”

said Levi Garraway, M.D., Ph. D., Chief Medical Officer and Head of Global Product Development.

Interim data from the RAINBOWFISH study was also presented, demonstrating Evrysdi’s safety and efficacy in newborns. The US Food and Drug Administration (FDA) granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi in pre-symptomatic babies with SMA under the age of two months in January.

Evrysdi has been used to treat over 5,000 people in clinical trials, compassionate use, and real-world settings. As part of a collaboration with the SMA Foundation and PTC Therapeutics, Roche is leading the clinical development of Evrysdi.

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