Friday, Jun 7, 2024
After 5 years of treatment, 91% of children were alive — without treatment, children with Type 1 SMA would not be expected to live past 2 years of age
96% of Evrysdi-treated children could swallow, 80% could feed without a feeding tube and 59% could sit without support for at least 30 seconds
Evrysdi is now approved in more than 100 countries with over 15,000 patients treated globally
South San Francisco, CA -- June 7, 2024 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile of Evrysdi® (risdiplam) in children with Type 1 spinal muscular atrophy (SMA) from the open-label extension of the pivotal FIREFISH study. By the end of year 5, 91% of children treated with Evrysdi were alive, 81% were alive without permanent ventilation and the majority were able to sit without support for at least 30 seconds (59%). At the end of year 5, seven children were able to stand, three with support, four unaided and six could walk with support. Without disease modifying treatment, natural history studies indicate that children with Type 1 SMA would not only never be able to reach such milestones, but also not typically live past the age of two. The data were presented at the Cure SMA Research & Clinical Care Meeting, June 5 - 7, 2024.
“These long-term findings confirm the ongoing benefit of Evrysdi for children with Type 1 SMA,” said Professor Giovanni Baranello, M.D., Ph.D., UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, U.K. “Children treated with Evrysdi over 5 years have maintained or improved their ability to sit, stand and walk — critical skills for development and daily living. An overwhelming majority also maintained the ability to swallow and to eat without a feeding tube.”
Motor function abilities, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) and Hammersmith Infant Neurological Examination 2 (HINE-2), were maintained or continued to be achieved in those treated with Evrysdi. The FIREFISH results showed most children treated with Evrysdi also maintained their feeding and swallowing abilities. Of those assessed at year 5, 96% were able to swallow and 80% were able to feed without a feeding tube.
“This is the final readout of the FIREFISH study, which has provided a wealth of insights and data, helping to firmly establish Evrysdi as an important treatment option, improving the lives of children across the globe living with SMA,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “This would not have been possible without the commitment and dedication of the children and families who participated, as well as numerous healthcare professionals and patient support organizations to whom we are immensely thankful.”
No treatment-related adverse events (AEs) led to treatment discontinuation or withdrawal from the study. The overall rate of AEs decreased by 66% between year 1 and the final year of study. The most common AEs were upper respiratory tract infection (64%), pyrexia (64%) and pneumonia (50%). Hospitalizations declined over the 5-year treatment period and 22% of children did not require hospitalization at all since beginning treatment with Evrysdi.
Evrysdi is the only oral, non-invasive small molecule SMA treatment designed to be systemically delivered to both the central nervous system (CNS) and peripheral tissues.
Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form either by feeding tube or by mouth.
Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues, as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core motor functions such as swallowing, speaking and breathing.
Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. 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 more than 100 countries, and the dossier is under review in a further 13 countries.
Evrysdi is currently being, or has been, evaluated in five global multicenter trials in people with SMA:
About SMA
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and is among the leading genetic causes 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.
What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.
Important Safety Information
These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.
You may report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.
Please see full Prescribing Information for additional Important Safety Information.
About Genentech in Neuroscience
Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.
Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.
About Genentech
Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.
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