{"id":55004,"date":"2026-04-24T12:41:13","date_gmt":"2026-04-24T12:41:13","guid":{"rendered":"https:\/\/www.europesays.com\/ch\/55004\/"},"modified":"2026-04-24T12:41:13","modified_gmt":"2026-04-24T12:41:13","slug":"novartis-secures-positive-eu-backing-for-sma-gene-therapy-itvisma","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/ch\/55004\/","title":{"rendered":"Novartis Secures Positive EU Backing for SMA Gene Therapy Itvisma"},"content":{"rendered":"<p>    <img fetchpriority=\"high\" decoding=\"async\" src=\"https:\/\/www.europesays.com\/ch\/wp-content\/uploads\/2026\/04\/d3083c06a0d4ea5be73c5655a5ae1803.png\" alt=\"medical research 15 \u00a9The U.S. National Archives\" loading=\"eager\" height=\"351\" width=\"640\" class=\"yf-lglytj  loaded\"\/> medical research 15 \u00a9The U.S. National Archives      <\/p>\n<p class=\"yf-1fy9kyt\">Novartis (NYSE:NVS) said the European Medicines Agency\u2019s Committee for Medicinal Products for Human Use has issued a favourable opinion recommending approval of Itvisma, an intrathecal gene therapy for spinal muscular atrophy (SMA).<\/p>\n<p class=\"yf-1fy9kyt\">The recommendation covers use in children aged two and above, as well as adolescents and adults with 5q SMA carrying a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene. The treatment is designed as a one-time fixed-dose therapy aimed at replacing the defective SMN1 gene.<\/p>\n<p class=\"yf-1fy9kyt\">The positive opinion is based on findings from the pivotal STEER trial, alongside supporting data from the Phase IIIb STRENGTH and Phase I\/II STRONG studies.<\/p>\n<p class=\"yf-1fy9kyt\">In the STEER study, Itvisma delivered a statistically significant improvement of 2.39 points on the Hammersmith Functional Motor Scale, compared with a 0.51-point gain in the control group, achieving a p-value of 0.0074. These benefits were maintained over a 52-week follow-up period.<\/p>\n<p class=\"yf-1fy9kyt\">\u201cItvisma met the primary endpoint showing motor function improvement versus placebo,\u201d said Tim Hagenacker of the University Hospital Essen. \u201cEven a 1-point difference in the HFSME can translate into tangible functional gains for individuals with SMA, such as the ability to grasp a pen.\u201d<\/p>\n<p class=\"yf-1fy9kyt\">The STEER trial included 75 patients treated with Itvisma and 51 patients receiving a sham procedure. Results from both STEER and STRENGTH have been published in Nature Medicine.<\/p>\n<p class=\"yf-1fy9kyt\">A final decision from the European Commission is expected within around two months following the committee\u2019s recommendation.<\/p>\n<p class=\"yf-1fy9kyt\">Novartis already markets Zolgensma, a gene therapy approved for infants and young children with SMA, positioning Itvisma as a potential expansion of its treatment offering across a broader patient population.<\/p>\n<p class=\"yf-1fy9kyt\">Novartis stock price<\/p>\n","protected":false},"excerpt":{"rendered":"medical research 15 \u00a9The U.S. National Archives Novartis (NYSE:NVS) said the European Medicines Agency\u2019s Committee for Medicinal Products&hellip;\n","protected":false},"author":2,"featured_media":55005,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":"","_share_on_mastodon":"0"},"categories":[127],"tags":[7758,212,30856,4875,206,30855,9913],"class_list":["post-55004","post","type-post","status-publish","format-standard","has-post-thumbnail","category-novartis","tag-european-medicines-agency","tag-gene-therapy","tag-hammersmith-functional-motor-scale","tag-medical-research","tag-novartis","tag-novartis-stock-price","tag-spinal-muscular-atrophy"],"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@ch\/116459738832194730","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/55004","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/comments?post=55004"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/55004\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media\/55005"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media?parent=55004"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/categories?post=55004"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/tags?post=55004"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}