{"id":31416,"date":"2022-06-01T01:58:02","date_gmt":"2022-06-01T01:58:02","guid":{"rendered":"https:\/\/harchi90.com\/roche-wins-new-evrysdi-approval-for-the-youngest-sma-patients-endpoints-news\/"},"modified":"2022-06-01T01:58:02","modified_gmt":"2022-06-01T01:58:02","slug":"roche-wins-new-evrysdi-approval-for-the-youngest-sma-patients-endpoints-news","status":"publish","type":"post","link":"https:\/\/harchi90.com\/roche-wins-new-evrysdi-approval-for-the-youngest-sma-patients-endpoints-news\/","title":{"rendered":"Roche wins new Evrysdi approval for the youngest SMA patients – Endpoints News"},"content":{"rendered":"
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Roche just turned the spinal muscular atrophy race up another notch.<\/p>\n

The Big Pharma company won another FDA approval for its oral SMA drug Evrysdi, though Tuesday’s green light comes for babies younger than two months old. It’s the first such approval for any SMA drug available to administer at home in this infant population.<\/p>\n

\u201cThe approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,\u201d Phase II principal investigator Richard Finkel said in a statement.<\/p>\n

First approved back in 2020 as the first oral SMA drug, Evrysdi quickly became a key player in the SMA field and a challenger to Biogen’s Spinraza and Novartis’ Zolgensma. The oral administration likely makes Evrysdi more appealing to patients, given Spinraza is injected into the spine and Zolgensma is given through IV infusion.<\/p>\n

Analysts have pegged peak Evrysdi sales at roughly $ 2 billion, making it a potential blockbuster despite being priced much lower than its competitors. One year of Evrysdi treatment maxes out at $ 340,000 per year, compared to Spinraza’s price tag of roughly $ 625,000 to $ 750,000 in the first year and $ 375,000 in subsequent years, and Zolgensma’s one-time cost of $ 2.1 million.<\/p>\n

For infants weighing less than 15 pounds and under two years old, Evrysdi costs are even smaller, clocking in at less than $ 100,000 annually. The pricing scale caps when the child reaches 44 pounds, which puts the average at about $ 7727.27 per pound per year for every patient.<\/p>\n

The lower costs haven’t stopped Evrysdi from becoming one of Roche’s quickest-growing cash engines, either. In the first quarter of 2022, the drug pulled in more than $ 235 million in sales, a 189% increase over the same time period last year. Roche said last month European markets saw the biggest uptake.<\/p>\n

Tuesday’s approval comes from interim results of an open-label Phase II study examining about 25 infants with confirmed SMA diagnoses who aren’t showing symptoms yet. Of the six babies evaluable for efficacy thus far, all were able to sit after one year of treatment, Roche said. Additionally, four of the six could stand, three could walk independently and all were alive without ventilation after one year.<\/p>\n

In addition to this study, dubbed RAINBOWFISH, Roche continues to test Evrysdi in four other clinical trials looking at both infants and adults. The drug, licensed by Roche from PTC Therapeutics, is designed to treat SMA by modifying how the SMN2 gene is spliced, leading to increased functional protein levels.<\/p>\n<\/p><\/div>\n","protected":false},"excerpt":{"rendered":"

Roche just turned the spinal muscular atrophy race up another notch. The Big Pharma company won another FDA approval for its oral SMA drug Evrysdi, though Tuesday’s green light comes for babies younger than two months old. It’s the first such approval for any SMA drug available to administer at home in this infant population. …<\/p>\n

Roche wins new Evrysdi approval for the youngest SMA patients – Endpoints News<\/span> Read More »<\/a><\/p>\n","protected":false},"author":1,"featured_media":31417,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"default","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","spay_email":"","jetpack_publicize_message":"","jetpack_is_tweetstorm":false,"jetpack_publicize_feature_enabled":true},"categories":[8],"tags":[],"jetpack_featured_media_url":"https:\/\/i0.wp.com\/endpts.com\/wp-content\/uploads\/2020\/08\/Roche-social-AP-scaled.jpg?ssl=1","jetpack_publicize_connections":[],"jetpack_sharing_enabled":true,"fifu_image_url":"https:\/\/endpts.com\/wp-content\/uploads\/2020\/08\/Roche-social-AP-scaled.jpg","_links":{"self":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts\/31416"}],"collection":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/comments?post=31416"}],"version-history":[{"count":0,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts\/31416\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/media\/31417"}],"wp:attachment":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/media?parent=31416"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/categories?post=31416"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/tags?post=31416"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}