Monday, May 30, 2022
Approval based on interim RAINBOWFISH data which show pre-symptomatic babies treated with Evrysdi for at least one year were able to sit, stand and walk
Prescribing information updated with FIREFISH data showing the majority of symptomatic babies treated with Evrysdi for at least two years could sit for at least 5 seconds
Evrysdi has proven efficacy in babies, children and adults with more than 5,000 patients treated to date
South San Francisco, CA -- May 30, 2022 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment. Evrysdi is now approved to treat SMA in children and adults of all ages.
Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. All infants were alive at 12 months without permanent ventilation.
“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” said Richard Finkel, M.D., RAINBOWFISH Principal Investigator and Director of the Experimental Neuroscience Program at St. Jude Children’s Research Hospital. “With the inclusion of SMA in newborn screening programs, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed.”
As part of the label extension, the Evrysdi prescribing information has also been updated to include recent two-year pooled data from Parts 1 and 2 of the FIREFISH study, which demonstrate long-term efficacy and safety in symptomatic infants with Type 1 SMA. The study enrolled babies aged 1-7 months and after two years of treatment with Evrysdi at the recommended dose (n=58), 60% of infants were able to sit without support* for 5 seconds, 40% for 30 seconds and 28% of infants were able to stand.** Without treatment, infants do not achieve these milestones in the natural history of the disease. There were no treatment-related adverse events leading to withdrawal. The most common adverse reactions were upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting and cough.
“The priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from pre-symptomatic newborns to older adults. We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers.”
Evrysdi is approved in 81 countries and the dossier is under review in a further 27 countries. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
* Bayley Scales of Infant and Toddler Development – Third Edition
** Hammersmith Infant Neurological Examination (HINE-2)
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 by mouth or by feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein in the central nervous system (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 movement.
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 81 countries and the dossier is under review in a further 27 countries.
Evrysdi is currently being evaluated in five 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 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 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.
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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