Therapy Areas: Central Nervous System
Genentech Touts New Evrysdi Data Demonstrating Long-Term Efficacy and Safety in a Broad Population of People with Spinal Muscular Atrophy
16 March 2022 - - US-based biotechnology company Genentech, a member of Switzerland's Roche Group (SIX: RO) (OTCQX: RHHBY) has released new data for Evrysdi (risdiplam) in spinal muscular atrophy, the company said.

Presentations included new three-year data from the SUNFISH study which further confirmed the long-term efficacy and safety of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA.

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 Clinical and Scientific Conference, March 13-16, 2022.

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 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 in SUNFISH decreased over three years, and a trend towards a lower rate of serious adverse events 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 ≥3 points) or stabilization (change ≥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 US Food and Drug Administration granted priority review of a supplemental new drug application 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.

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

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system and peripheral tissues as demonstrated in animal models.

SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

Evrysdi was granted PRIME designation by the European Medicines Agency in 2018 and Orphan Drug Designation by the US Food and Drug Administration in 2017.

In 2021 Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research award for Drug Discovery. Evrysdi is currently approved in 76 countries and the dossier is under review in a further 29 countries.

Evrysdi is currently being evaluated in five multicenter trials in people with SMA.
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