Sunday, Sep 27, 2020
Exploratory efficacy data showed 88% of infants treated with Evrysdi were alive and did not require permanent ventilation at two years
59% of infants were able to sit without support for at least 5 seconds
No new safety signals were identified
In August, the FDA approved Evrysdi for the treatment of SMA in adults and children 2 months and older
South San Francisco, CA -- September 27, 2020 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve and achieve motor milestones.
This exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. In addition, at two years, 59% (10/17 vs. 7/17 at 1-year) of infants were able to sit without support for at least 5 seconds, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III). Sixty-five percent (11/17 vs. 9/17 at 1-year) had maintained upright head control, 29% (5/17 vs. 2/17 at 1-year) could turn themselves over and 30% (5/17 vs. 1/17 at 1-year) were able to stand either supporting weight or with support. After two years of treatment with Evrysdi, 71% (12/17 vs. 10/17 at 1-year) of infants achieved a CHOP-INTEND* score of 40 points or more and all infants increased their score from month 12 to month 24. Of the infants alive at two years (n=14), 100% maintained the ability to swallow and 93% (13/14) were able to feed orally. Safety for Evrysdi in the FIREFISH study was consistent with its previously reported safety profile and no new safety signals were identified. The most common adverse events (n=21) included fever (pyrexia; 71%), upper respiratory tract infection (52%), cough (33%), vomiting (33%), diarrhea (29%) and respiratory tract infection (29%). The most serious adverse event that occurred in 24% of infants was pneumonia.
“We are highly encouraged by the results we are seeing in the second year of treatment with Evrysdi,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “These results build on the efficacy and safety demonstrated by Evrysdi in pivotal trials, and we look forward to continued assessments of both survival and motor function during long-term follow up for this first-of-its-kind treatment.”
At the time of the analysis, the youngest infant was 28.4 months and the oldest was 45.1 months of age. The median age at enrollment was 6.3 months. Of the 17 infants treated with the therapeutic dose, two experienced fatal complications of their disease at 8 and 13 months of treatment and one infant was withdrawn from the study and sadly died 3.5 months later. None of these were attributed by the investigator as related to Evrysdi.
The data were presented at the virtual 25th International Annual Congress of the World Muscle Society.
Evrysdi is being studied in more than 450 people as part of a broad and robust clinical trial program in SMA, with people ranging from birth to 60 years old, and includes pre-symptomatic people and those previously treated with other SMA-targeting therapies. Evrysdi is designed to treat SMA by increasing and sustaining the 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. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
*Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders
About Evrysdi™ (risdiplam)
Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
The U.S. Food and Drug Administration (FDA) approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older. Risdiplam was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. At this time, Evrysdi has been filed in 16 international markets: Australia, Brazil, Chile, India, Indonesia, Israel, Kuwait, Macedonia, Malaysia, Russia, Singapore, South Korea, Taiwan, Thailand, Ukraine and the United Arab Emirates. In addition, four health authorities worldwide are currently reviewing the application: Canada, China, EU and Switzerland.
Risdiplam is currently being evaluated in four multicenter trials in people with 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.
What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
It is not known if Evrysdi is safe and effective in children under 2 months of age.
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, patients should ask their healthcare provider or pharmacist.
Patients may report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. Patients may also report side effects to Genentech at 1-888-835-2555.
Please see the full Prescribing Information for additional Important Safety Information.
About Genentech in Neuroscience
Neuroscience is a major focus of research and development at Genentech and Roche. 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, stroke, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, Duchenne muscular dystrophy and autism spectrum disorder. 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.
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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