Risdiplam

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi ® (risdiplam) tablet for people living with spinal muscular atrophy (SMA). Evrysdi is the only non-invasive disease-modifying treatment for SMA. The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water. “Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.” The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking water (e.g., filtered water), and original oral solution provide comparable exposure to risdiplam. This means patients who take the tablet can expect the same established efficacy and safety as the Evrysdi oral solution. The Evrysdi oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution. “We cannot underestimate the value that comes with simplifying treatment administration and disease management for people who are living with SMA or those caring for them,” said Kenneth Hobby, President of Cure SMA. “This new room temperature stable formulation option offers an additional choice that may more conveniently fit into daily living activities such as working, traveling, and education.” As part of the label extension, the Evrysdi prescribing information has been updated to include guidance on tablet administration and storage. The new tablet, expected to be available in the coming weeks, is suitable for people 2 years of age or older who weigh more than 44 lbs (20 kgs). 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 ) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home or on the go, either in liquid form (by feeding tube or by mouth) or in the form of a tablet (by mouth only, swallowed whole or dispersed in water). Evrysdi is designed to treat SMA by increasing and sustaining the production of 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 core functions. 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 (FDA) 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, with more than 16,000 people with SMA treated globally. Evrysdi is currently being, or has been, evaluated in numerous global multicenter trials in people with SMA: FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately 5.5 months of age (median) at the time of enrollment and of the 58 infants that completed the first year of treatment, 52 entered the open-label extension study. The study met its primary endpoint and has concluded after 5 years of follow up. SUNFISH (NCT02908685) – a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. The study met its primary endpoint and has concluded after 5 years of follow up. JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies prior to receiving Evrysdi. The study has completed recruitment (n=174). RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies (n=26), from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study met its primary endpoint. MANATEE (NCT05115110) – a Phase II/III clinical study to evaluate the safety and efficacy of GYM329 (RG6237), an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2 to 10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021. The study is currently active. HINALEA 1 (NCT05861986) and HINALEA 2 (NCT05861999) – Phase IV clinical studies to evaluate the effectiveness and safety of Evrysdi in patients under 2 years of age at enrollment, who received onasemnogene abeparvovec gene therapy either pre-symptomatically or post-symptomatically, following a genetically confirmed diagnosis of 5q–autosomal recessive SMA. The studies are currently recruiting. PUPFISH (NCT05808764) – a Phase II, open-label study to investigate the pharmacokinetics and safety of Evrysdi in babies with SMA who are under 20 days of age (at first dose). The study is currently recruiting. About SMA SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one 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 Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you: are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine are a woman who can become pregnant: Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting http://www.evrysdipregnancyregistry.com are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby Tell your healthcare provider about all the medicines you take If you were prescribed Evrysdi for Oral Solution, you should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water The most common side effects of Evrysdi include: For later-onset SMA: fever diarrhea rash For infantile-onset SMA: fever diarrhea rash runny nose, sneezing, and sore throat (upper respiratory infection) lung infection (lower respiratory infection) constipation vomiting cough 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. For more information, go to https://www.evrysdi.com/ . 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 . All trademarks used or mentioned in this release are protected by law.