- New data show pre-symptomatic babies with spinal muscular atrophy (SMA) treated with Evrysdi maintained the ability to swallow
- Evrysdi has demonstrated consistent clinically meaningful efficacy in adults, children, and babies two months and older and is now approved in 58 countries worldwide
- Further presentations included data from studies supporting the efficacy, safety, and durability of gene therapy, SRP-9001, in the treatment of Duchenne muscular dystrophy (DMD)
“These new data for Evrysdi may help extend the potential benefits of this medicine to the youngest SMA patients. Also, the data from SRP-9001 have helped to optimise the design of the upcoming Phase III trial for DMD,” said
At WMS 2021, data from the ongoing open label RAINBOWFISH study were presented. Four out of five of those treated with Evrysdi for at least 12 months achieved standing and walking independently within the
Previously reported results showed that babies treated with Evrysdi for at least 12 months achieved Hammersmith Infant Neurological Examination (HINE-2) motor milestones, with 100% (n=5) able to maintain head control, sitting upright, rolling and crawling.
These data further add to the growing body of evidence supporting Evrysdi’s efficacy in a broad patient population. More than 4,000 patients have been treated with Evrysdi in clinical trials, compassionate use, and real-world settings.
In DMD, three-year data from the open-label trial, Study SRP-9001-101, evaluating the safety of a single dose of the investigational gene therapy SRP-9001 in four ambulatory children aged 4 and 7 years old with DMD, were presented. The study showed that SRP-9001 was well-tolerated with key functional assessment, measured by the North Star Ambulatory Assessment (NSAA), demonstrating an overall improvement in motor ability compared to baseline. The improvements in motor abilities were maintained over three years, signifying a durable response. The results support the continuation of clinical investigation to further assess the benefit/risk of SRP-9001 in patients with DMD.
In addition, results from Part 1 of Study SRP-9001-102, an ongoing, randomised, double-blind, placebo-controlled clinical trial evaluating the safety, efficacy, and tolerability of a single dose of SRP-9001 in 41 boys with DMD, showed that the study met its primary biological endpoint of change in micro-dystrophin protein expression from baseline. Participants treated with SRP-9001, generally showed an increase in NSAA total score compared to placebo at 48 weeks, although this increase did not achieve statistical significance compared to that of patients
The results of these studies provide important information for SRP-9001’s ongoing clinical development programme, with outcomes from Study 101 and 102 informing the design of the Phase 3 trial for SRP-9001, due to commence globally by the end of the year.
About Evrysdi® (risdiplam)
Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA by increasing 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
About SMA
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.
About SRP-9001
SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) is an investigational gene therapy designed to deliver the micro-dystrophin-encoding gene directly to the skeletal and cardiac muscle for the targeted production of the micro-dystrophin protein to enable a durable clinical response. Sarepta Therapeutics is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in
About DMD
DMD is a rare X-linked, progressive neuromuscular disease caused by mutations in the DMD gene that disrupts the production of functional dystrophin protein, leading to a loss of muscle function and premature death. DMD is one of the most common fatal genetic disorders, affecting approximately one in every 3,500 to 5,000 male births worldwide.
Symptoms usually appear in infants and toddlers, with affected children presenting developmental delays such as difficulty walking, climbing stairs or standing from a sitting position. As DMD progresses, muscle weakness involves the arms, trunk, and other areas, meaning patients often require full-time use of a wheelchair in their early teens. Longevity is limited due to cardiac and/or respiratory failure.
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