Genentech : Data On Evrysdi Shows Improvement Of Motor Function In Spinal Muscular Atrophy Patients

(RTTNews) - Genentech's, a member of the Roche Group (RHHBY), Evrysdi or risdiplam continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with type 2 or type 3 spinal muscular atrophy, as per 2-year longer-term data from Part 2 of SUNFISH study.

The company said the study suggested 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.

According to the company, 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.

The most common adverse events observed in the Evrysdi arm and the placebo and Evrysdi arm from 12-24 months were upper respiratory tract infection, nasopharyngitis, pyrexia, headache, diarrhea, vomiting and cough. The most common serious adverse events were pneumonia and influenza.

Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat spinal muscular atrophy by increasing and sustaining production of the survival of motor neuron or SMN protein. Survival of motor neuron 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 spinal muscular atrophy in adults and children 2 months of age and older in August of 2020. Spinal muscular atrophy 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. Spinal muscular atrophy is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein.

The 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.

Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.