Latest news with #Evrysdi
Yahoo
6 days ago
- Business
- Yahoo
Roche's Evrysdi tablet approved by European Commission as first and only for Spinal Muscular Atrophy (SMA)
Simplified storage and administration of new tablet formulation may provide greater freedom and independence for people with SMA Evrysdi offers the same efficacy and safety demonstrated in available oral solution Evrysdi is the only non-invasive disease-modifying SMA treatment, with more than 18,000 people with SMA treated globally to date Basel, 04 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved a label extension for Evrysdi® (risdiplam) to include a new, room-temperature stable tablet for people living with spinal muscular atrophy (SMA). The 5mg tablet (approx. 6.5mm), which can either be swallowed whole or dispersed in water, can be taken with or without food and does not require refrigeration, when stored at room temperature. Administered at home, Evrysdi is the only non-invasive disease modifying treatment available for people living with SMA. 'The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. 'With over 18,000 people treated to date, Evrysdi's proven efficacy, safety and convenience has significantly improved the course of disease for people living with SMA.' Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein throughout the entire central nervous system (CNS) and in peripheral tissues. Together with this innovative mode of action, the new tablet formulation offers additional portability and convenience benefits for the thousands of people living with SMA, their families and caregivers. 'We welcome the development of new treatment formulations that have the potential to further simplify disease management and care for people living with SMA,' Nicole Gusset, Chief Executive Officer, SMA Europe commented. 'This is a disease requiring daily management, and it is paramount that people living with SMA, and those who care for them, are given options to optimise treatment administration.' The approval is based on data from a bioequivalence study (NCT04718181) evaluating the 5mg tablet formulation of Evrysdi, which can either be swallowed whole or dispersed in water. Results presented at SMA Europe's 4th Scientific International Congress in 2024 demonstrated that the tablet formulation and original oral solution provided bioequivalence to Evrysdi, meaning individuals taking the tablet can expect the same established efficacy and safety as the oral solution. The 5mg tablet formulation is suitable for people two years of age or older, who weigh 20kg (44 lbs) or more and are able to swallow without the use of a feeding tube. The original oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution. Roche 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 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, which can either be swallowed whole or dispersed in water. Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues. 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 18,000 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 five 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 2-10 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 recruiting. 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 SMASMA 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 Roche in NeuroscienceNeuroscience is a major focus of research and development at 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. Roche is 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 RocheFounded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit All trademarks used or mentioned in this release are protected by law. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr Rebekka SchnellPhone: +41 79 205 27 03 Attachment Media Release Evrysdi Tablet EC Approval EnglishError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
6 days ago
- Business
- Yahoo
Roche's Evrysdi tablet approved by European Commission as first and only for Spinal Muscular Atrophy (SMA)
Simplified storage and administration of new tablet formulation may provide greater freedom and independence for people with SMA Evrysdi offers the same efficacy and safety demonstrated in available oral solution Evrysdi is the only non-invasive disease-modifying SMA treatment, with more than 18,000 people with SMA treated globally to date Basel, 04 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved a label extension for Evrysdi® (risdiplam) to include a new, room-temperature stable tablet for people living with spinal muscular atrophy (SMA). The 5mg tablet (approx. 6.5mm), which can either be swallowed whole or dispersed in water, can be taken with or without food and does not require refrigeration, when stored at room temperature. Administered at home, Evrysdi is the only non-invasive disease modifying treatment available for people living with SMA. 'The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. 'With over 18,000 people treated to date, Evrysdi's proven efficacy, safety and convenience has significantly improved the course of disease for people living with SMA.' Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein throughout the entire central nervous system (CNS) and in peripheral tissues. Together with this innovative mode of action, the new tablet formulation offers additional portability and convenience benefits for the thousands of people living with SMA, their families and caregivers. 'We welcome the development of new treatment formulations that have the potential to further simplify disease management and care for people living with SMA,' Nicole Gusset, Chief Executive Officer, SMA Europe commented. 'This is a disease requiring daily management, and it is paramount that people living with SMA, and those who care for them, are given options to optimise treatment administration.' The approval is based on data from a bioequivalence study (NCT04718181) evaluating the 5mg tablet formulation of Evrysdi, which can either be swallowed whole or dispersed in water. Results presented at SMA Europe's 4th Scientific International Congress in 2024 demonstrated that the tablet formulation and original oral solution provided bioequivalence to Evrysdi, meaning individuals taking the tablet can expect the same established efficacy and safety as the oral solution. The 5mg tablet formulation is suitable for people two years of age or older, who weigh 20kg (44 lbs) or more and are able to swallow without the use of a feeding tube. The original oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution. Roche 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 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, which can either be swallowed whole or dispersed in water. Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues. 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 18,000 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 five 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 2-10 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 recruiting. 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 SMASMA 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 Roche in NeuroscienceNeuroscience is a major focus of research and development at 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. Roche is 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 RocheFounded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit All trademarks used or mentioned in this release are protected by law. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr Rebekka SchnellPhone: +41 79 205 27 03 Attachment Media Release Evrysdi Tablet EC Approval EnglishError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
01-03-2025
- Health
- Yahoo
Buckhannon man raises awareness on Rare Disease Day
BUCKHANNON, (WBOY) — Every year since 2008, Rare Disease Day has been held on the last day of February to raise awareness about the research that goes into rare diseases like spinal muscular atrophy (SMA). SMA is a progressive neuromuscular disorder that affects functions like breathing, swallowing and walking. According to the SMA Foundation, there are 10,000-25,000 children and adults in the U.S. with the disease. 12 News spoke with Bryan Samples, a Buckhannon man who was diagnosed with SMA at 18 months old, and despite the disease creating hurdles in his life, he's been able to graduate, get married and live his life to the fullest. With all of these accomplishments, Samples has a message of hope for others like him. 'If you want something, go after it, never think that you're less than anybody else. You can lead a normal life and you can do anything anybody else can do, you just might have to do it in a different way,' Bryan 'James' Samples, Advocate for Rare Disease Awareness said. Mister Bee announces 'Mothman' flavored potato chips Samples said he's looking forward to a world where there is a cure for SMA, thanks to advancements in medication Evrysdi, which he's been taking for a while now and has seen changes. 'As long as things keep on getting better, you know, in the future we might not even know that someone has spinal muscular atrophy and that's pretty amazing,' To learn more about the medication, you can go here. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Yahoo
27-02-2025
- Health
- Yahoo
Fetus Receives Life-Saving Medication Inside Womb in Medical First
Doctors in the US have treated a fetus with a rapidly progressive congenital disease from inside the womb for the very first time. Instead of giving medicine to a child with severe neuromuscular complications after they are born, a new case study details how a prospective mother agreed to take the medicine for her developing fetus while she was still pregnant. A prenatal test had shown that her fetus held two genetic mutations indicating type 1 spinal muscular atrophy (SMA), which usually results in severe muscle weakness and breathing difficulties within six months of birth. Historically, most children with type 1 SMA die by their second birthday, usually from respiratory failure. However, the child in this study has so far made it to the age of two and a half years with no sign of symptoms. The parents of the treated child had previously experienced the loss of their baby to spinal muscular atrophy, and when testing showed their most recent fetus also had the progressive neuromuscular disease, they wanted to know if they could start treatments earlier. For this single case, the FDA approved the early administration of the oral drug risdiplam, brand name Evrysdi, owned by the pharmaceutical company F. Hoffmann-La Roche AG. Over the years, randomized controlled trials have shown risdiplam to be both safe and effective at treating spinal muscular atrophy in newborns, and the younger the kids are when they start receiving the medicine, the better the outcomes overall. In the new study, the pregnant mother took a dose of risdiplam each day for six weeks before giving birth. Testing found that the medicine trickled right through her umbilical cord blood and the amniotic fluid bathing her fetus. After birth, it was the infant that was given the daily oral drug, not the mother. Pediatric neurologist Michelle Farrar, who is working on a minimally invasive gene therapy treatment for SMA in Australia, told Smriti Mallapaty at Nature that the baby girl with SMA "has been effectively treated, with no manifestations of the condition" even 30 months after birth. In 2020, risdiplam was first approved by the US Food and Drug Administration (FDA) for kids with spinal muscular atrophy, but only over the age of two months. Recent clinical trials funded by Roche, however, found the majority of children with spinal muscular atrophy treated with risdiplam before six weeks of age could swallow, feed, sit, stand, and walk on their own after two years of treatment. None required permanent ventilation. Risdiplam works to prevent the degeneration of nerve cells in the brain and body by increasing the concentration of a crucial protein, called survival motor neuron (SMN) protein. Active SMN proteins are lacking in those with genetic mutations that cause spinal muscular atrophy. "In children with SMA, motor neuron degeneration starts before the onset of symptoms, so time is of the essence if we hope to preserve muscle function," said pediatric neurologist Laurent Servais in a press release for a Roche clinical trial in 2024. "It's heartening to see that through early intervention with Evrysdi these children have achieved important milestones like sitting, standing, and walking that would typically be unattainable without treatment." Whether or not the recent case study in the US will inspire and embolden doctors and parents to trial risdiplam at earlier stages of development remains to be seen. "Results in this single case cannot be generalized but may support the consideration of prenatal risdiplam treatment for SMA identified in utero," write the authors of the correspondence. The study was published in The New England Journal of Medicine. There's a Critical Thing We Can All Do to Keep Alzheimer's Symptoms at Bay Traces of a Nutrient in Brazil Nuts Could Fight Cancer, But Dosage Is Crucial Tardigrade Protein Could Soon Make Cancer Patients More Radiation Proof
Associated Press
28-01-2025
- Business
- Associated Press
Royalty Pharma to Announce Fourth Quarter and Full Year 2024 Financial Results on February 11, 2025
NEW YORK, Jan. 28, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it will report its fourth quarter and full year 2024 financial results on Tuesday, February 11, 2025 before the U.S. financial markets open. The company will host a conference call and simultaneous webcast at 8:30 a.m. Eastern Time that day. Conference Call Information Please visit the 'Investors' page of the company's website at to obtain conference call information and to view the live webcast. A replay of the conference call and webcast will be archived on the company's website for at least 30 days. About Royalty Pharma Founded in 1996, Royalty Pharma is the largest buyer of biopharmaceutical royalties and a leading funder of innovation across the biopharmaceutical industry, collaborating with innovators from academic institutions, research hospitals and non-profits through small and mid-cap biotechnology companies to leading global pharmaceutical companies. Royalty Pharma has assembled a portfolio of royalties which entitles it to payments based directly on the top-line sales of many of the industry's leading therapies. Royalty Pharma funds innovation in the biopharmaceutical industry both directly and indirectly – directly when it partners with companies to co-fund late-stage clinical trials and new product launches in exchange for future royalties, and indirectly when it acquires existing royalties from the original innovators. Royalty Pharma's current portfolio includes royalties on more than 35 commercial products, including Vertex's Trikafta, GSK's Trelegy, Roche's Evrysdi, Johnson & Johnson's Tremfya, Biogen's Tysabri and Spinraza, AbbVie and Johnson & Johnson's Imbruvica, Astellas and Pfizer's Xtandi, Novartis' Promacta, Pfizer's Nurtec ODT and Gilead's Trodelvy, and 14 development-stage product candidates. For more information, visit
