{"id":54970,"date":"2023-03-20T07:05:13","date_gmt":"2023-03-20T06:05:13","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/new-four-year-data-for-genentechs-evrysdi-reinforce-long-term-efficacy-and-safety-profile-in-some-of-the-most-severely-affected-people-with-types-2-and-3-spinal-muscular-atrophy-sma\/"},"modified":"2023-03-20T07:05:13","modified_gmt":"2023-03-20T06:05:13","slug":"new-four-year-data-for-genentechs-evrysdi-reinforce-long-term-efficacy-and-safety-profile-in-some-of-the-most-severely-affected-people-with-types-2-and-3-spinal-muscular-atrophy-sma","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/new-four-year-data-for-genentechs-evrysdi-reinforce-long-term-efficacy-and-safety-profile-in-some-of-the-most-severely-affected-people-with-types-2-and-3-spinal-muscular-atrophy-sma\/","title":{"rendered":"New Four-Year Data for Genentech\u2019s Evrysdi Reinforce Long-Term Efficacy and Safety Profile in Some of the Most Severely Affected People With Types 2 and 3 Spinal Muscular Atrophy (SMA)"},"content":{"rendered":"
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\n\u2013 <\/i>Data from pivotal SUNFISH study showed increases in motor function observed during the first year were maintained through the fourth year, while the overall rate of adverse events continued to decrease <\/i>\u2013<\/i>\n<\/p>\n

\n\u2013 <\/i>Data confirm long-term efficacy and safety profile of Evrysdi in a broad range of people with Type 2 and non\u2011ambulant Type 3 SMA <\/i>\u2013<\/i>\n<\/p>\n

\n\u2013 <\/i>More than 8,500 people\u2014from newborns to those over 60\u2014have been treated with Evrysdi, which is now approved in more than 90 countries worldwide <\/i>\u2013<\/i>\n<\/p>\n

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new long-term data for Evrysdi\u00ae<\/sup> (risdiplam) in a broad range of people aged 2-25 years with spinal muscular atrophy (SMA) from the pivotal SUNFISH study. Data confirm increases in motor function were sustained at four years and the overall rate of adverse events continued to decrease over the 48-month period, reinforcing the long-term efficacy and safety of Evrysdi. Participants also reported continuous improvement or stabilization when independently performing activities of daily living such as picking up and moving objects. The data were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 19-22, 2023.\n<\/p>\n

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\n\u201cPreserving long-term independence and the ability to perform daily tasks is an important measure for people living with SMA and their caregivers. It\u2019s encouraging to see that Evrysdi has meaningfully affected this aspect of their lives,\u201d said Laurent Servais, M.D., Ph.D., Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre. \u201cThis trial included people with Type 2 and 3 SMA, including patients with advanced disease. These latest data make us confident that the improvement observed during the first year of treatment is sustained during four years which contrasts with the decline we would observe in the absence of treatment.\u201d\n<\/p>\n

\nThe increase in motor function from baseline observed during the first year of the study was maintained through the fourth year of treatment with Evrysdi, as measured by changes in Motor Function Measure 32 (MFM-32) and Revised Upper Limb Module (RULM). Without treatment, natural history data show that patients with Type 2 or 3 SMA typically show a decline in motor function over time. Evrysdi was well tolerated over the four-year time period. Adverse events (AEs) and serious adverse events (SAEs) were reflective of the underlying disease. The most commonly reported AEs include headache, fever (pyrexia) and upper respiratory tract infection. No treatment-related AEs led to withdrawal from the study.\n<\/p>\n

\n\u201cThese new data show that treatment with Evrysdi may help people with Type 2 or 3 SMA to maintain improvements in muscle strength and mobility over the course of several years,\u201d said Levi Garraway, M.D., Ph.D., Genentech\u2019s chief medical officer and head of Global Product Development. \u201cThe longer-term results of this study add to the robust body of findings from our broad clinical trial program evaluating Evrysdi across a variety of ages, disease severities and treatment histories.\u201d\n<\/p>\n

\nIn addition to bringing Evrysdi to people around the world, Genentech also leads its clinical development as part of a collaboration with the SMA Foundation and PTC Therapeutics. Genentech is currently investigating Evrysdi in combination with an anti-myostatin molecule targeting muscle growth in the Phase II\/III trial MANATEE for the treatment of SMA.\n<\/p>\n

\nAbout Evrysdi\u00ae<\/sup> (risdiplam)<\/b>\n<\/p>\n

\nEvrysdi<\/a> 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.\n<\/p>\n

\nEvrysdi 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 movement.\n<\/p>\n

\nEvrysdi 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 90 countries and the dossier is under review in a further 16 countries.\n<\/p>\n

\nEvrysdi is currently being evaluated in five multicenter trials in people with SMA:\n<\/p>\n