RARE Daily

Genentech Reports Positive New Data for Evrysdi in SMA Type 2 or Type 3

March 16, 2021

Rare Daily Staff

Genentech, a member of the Roche Group, reported new exploratory 2-year longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi in people aged 2-25 years with Type 2 or non-ambulant Type 3 spinal muscular atrophy.

Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA by increasing and sustaining production of the survival of motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube. The U.S. Food and Drug Administration approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older in August of 2020.

The study suggests that gains in motor function observed with Evrysdi treatment at month 12 continued to improve or were maintained at month 24 across primary and secondary endpoint measures. Based on the natural history of the disease, people with Types 2 and 3 SMA who remain untreated decline in motor function over time. These data will be presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference taking place from March 15-18.

Patients in SUNFISH Part 2 ranged in age from 2-25 and were treated with Evrysdi (n=120) or placebo and Evrysdi (n=60; patients in the placebo arm received placebo for 12 months followed by Evrysdi treatment for 12 months). The study evaluated a number of exploratory 24-month endpoints, which provide important insights into motor function and its impact on daily life. Findings demonstrated that Evrysdi maintained motor function improvements between months 12 and 24 as measured by Motor Function Measure (MFM-32); increased motor function as measured by Revised Upper Limb Module (RULM), and the Hammersmith Functional Motor Scale-Expanded (HFMSE) between months 12 and 24; stabilized motor function for patients who began treatment with Evrysdi after 12 months of placebo as measured by MFM-32, RULM and HFMSE; and increased total score change from baseline, as measured by the caregiver-reported SMAIS upper limb module, and the patient-reported SMAIS score stabilized between months 12 and 24.

“These encouraging results confirm that the efficacy and safety of Evrysdi in people with Type 2 and Type 3 SMA can be sustained over time,” said Levi Garraway, chief medical officer and head of Global Product Development. “Therefore, these findings further highlight the potential longer-term benefit this first-of-its-kind medicine can have for people of varying ages and levels of SMA disease severity.”

Decreases in serious adverse events, high-grade adverse events and treatment-related adverse events were observed in the second year versus the first year in both treatment arms.

Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. At this time, Evrysdi has been approved in seven countries and submitted in 57, including the EU 27 and Norway and Iceland.

Photo: Levi Garraway, chief medical officer and head of Global Product Development, Genentech

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