{"id":49517,"date":"2022-10-12T07:01:49","date_gmt":"2022-10-12T05:01:49","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/positive-new-data-for-genentechs-evrysdi-in-largest-trial-ever-undertaken-in-patients-with-previously-treated-spinal-muscular-atrophy-sma\/"},"modified":"2022-10-12T07:01:49","modified_gmt":"2022-10-12T05:01:49","slug":"positive-new-data-for-genentechs-evrysdi-in-largest-trial-ever-undertaken-in-patients-with-previously-treated-spinal-muscular-atrophy-sma","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/positive-new-data-for-genentechs-evrysdi-in-largest-trial-ever-undertaken-in-patients-with-previously-treated-spinal-muscular-atrophy-sma\/","title":{"rendered":"Positive New Data for Genentech\u2019s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)"},"content":{"rendered":"
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\n\u2013 <\/i>New two-year Evrysdi data show improvement or maintenance of motor function in people with SMA, a progressive neuromuscular disease that can be fatal <\/i>\u2013<\/i>\n<\/p>\n

\n\u2013 <\/i>The JEWELFISH study enrolled the broadest and most diverse patient population ever studied in an SMA trial <\/i>\u2013<\/i>\n<\/p>\n

\n\u2013 <\/i>Longer-term safety data consistent with that previously seen in earlier trials and low study drop-out rate <\/i>\u2013<\/i>\n<\/p>\n

\n\u2013 <\/i>Evrysdi has proven efficacy in babies, children and adults, with more than 7,000 patients treated to date 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 two-year data from the JEWELFISH study evaluating Evrysdi\u00ae<\/sup> (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 months to 60 years at time of enrollment. Patients had been previously treated with other approved or investigational SMA-targeting therapies, including nusinersen (Spinraza\u00ae<\/sup>) or onasemnogene abeparvovec (Zolgensma\u00ae<\/sup>). Data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels which were sustained after two years of treatment. These data will be presented at the 27th<\/sup> World Muscle Society (WMS) congress, October 11-15, 2022.\n<\/p>\n

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\n\u201cThe consistent safety profile and exploratory efficacy we have seen in the JEWELFISH study, the largest ever conducted in previously treated patients, reinforces Evrysdi as a meaningful treatment option across SMA populations,\u201d said Dr. Claudia Chiriboga, Professor of Neurology and Pediatrics, Department of Neurology, Columbia University Medical Center, New York. \u201cThe findings add to our confidence when making treatment decisions for previously-treated patients in need.\u201d\n<\/p>\n

\nThe JEWELFISH study enrolled the broadest and most diverse patient population ever studied in an SMA trial. Of the 174 people enrolled, 36% (n=63) were adults, 63% (n=105) had a Hammersmith Functional Motor Scale Expanded (HFMSE) score of less than 10 at baseline, meaning their disease was very severe, and 83% (n=139) had scoliosis. Forty-four percent (n=76) of those enrolled had previously been treated with nusinersen (Spinraza), 41% (n=71) with olesoxime*, 8% (n=14) with onasemnogene abeparvovec (Zolgensma) and 7% (n=13) with RG7800*.\n<\/p>\n

\nPeople with SMA are unable to produce enough survival motor neuron (SMN) protein, leading to debilitating and potentially fatal muscle weakness. The study showed Evrysdi led to a two-fold increase in median SMN protein levels versus baseline after four weeks of treatment in all patient groups, irrespective of previous treatment. The SMN protein levels achieved after four weeks of treatment were maintained for over two years.\n<\/p>\n

\nObserved through exploratory efficacy endpoints, the study also suggests maintenance of motor function was sustained at two years of treatment as measured by change from baseline in Motor Function Measure 32 (MFM-32), Revised Upper Limb Module (RULM) and HFMSE total scores compared to the natural history of SMA in untreated patients. A recent survey conducted by patient advocacy group SMA Europe showed that more than 96% of people with SMA viewed disease stabilization as progress in terms of their expectations of treatment.\n<\/p>\n

\n\u201cThese important data demonstrate the safety and efficacy of Evrysdi in a broad, real-world population of people previously treated with an SMA-targeting therapy,\u201d said Levi Garraway, M.D., Ph.D., Genentech\u2019s chief medical officer and head of Global Product Development. \u201cThose enrolled in JEWELFISH had very severe disease, with over 80% having scoliosis, so maintaining motor function\u2013especially for a progressive disease\u2013can be potentially life-changing.\u201d\n<\/p>\n

\nThe overall adverse event (AE) and serious adverse event (SAE) profiles observed with Evrysdi treatment in JEWELFISH were reflective of underlying disease. The rate of AEs decreased by more than 50% between the first and second six-month period, and then remained stable thereafter. The rate of SAEs, including pneumonia, decreased throughout the 24-month period, with a total reduction of more than 50% by the second year. The most common AEs (reported in \u226512% of all patients; n=173) were pyrexia (24%), upper respiratory tract infection (21%), headache (18%), nasopharyngitis (16%), diarrhea (14%), nausea (13%) and cough (12%). The most common SAEs (reported in >2% of all patients) were pneumonia (3%), respiratory failure (2%), respiratory distress (2%), lower respiratory tract infection (2%) and upper respiratory tract infection (2%). The most common AEs\/SAEs were consistent with those observed in treatment-na\u00efve patients in our other three trials. Low rates of discontinuation from the study were observed, with a 5% rate per year over the 24-month period.\n<\/p>\n

\nGenentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.\n<\/p>\n

\n*RG7800 and olesoxime are no longer in development as investigational treatments for patients with SMA.<\/i>\n<\/p>\n

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

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

\nEvrysdi 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.\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 91 countries and the dossier is under review in a further 18 countries.\n<\/p>\n

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