{"id":908291,"date":"2026-09-28T07:18:08","date_gmt":"2026-09-28T07:18:08","guid":{"rendered":"https:\/\/www.newsbeep.com\/au\/908291\/"},"modified":"2026-09-28T07:18:08","modified_gmt":"2026-09-28T07:18:08","slug":"us-fda-approves-mirums-drug-for-rare-bone-disorder","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/au\/908291\/","title":{"rendered":"US FDA approves Mirum\u2019s drug for rare bone disorder"},"content":{"rendered":"<p class=\"text-primary article-summary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">WASHINGTON &#8211; The US FDA on Sept 25 approved Mirum Pharmaceuticals\u2019 pill for <a href=\"https:\/\/www.straitstimes.com\/singapore\/medical-mysteries-the-student-who-is-slowly-turning-into-stone?ref=inline-article\" rel=\"noopener nofollow\" class=\"gap-x-04 items-center inline text-primary-60 select-auto\" aria-label=\"link\" target=\"_blank\" data-testid=\"custom-link\">a rare bone disorder,<\/a> offering patients a new treatment option that can help slow the disease\u2019s progression.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">The once-daily drug zilurgisertib, branded as Atebrioz, is approved for patients aged 12 years and older with fibrodysplasia ossificans progressiva (FOP), which causes muscles, tendons and ligaments to gradually transform into bone, restricting movement and leading to severe disability.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">Atebrioz blocks ALK2, a protein that is abnormally active in most patients with FOP and drives bone formation outside the skeleton. The recommended dose is 100 milligrams.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">FOP is typically diagnosed in early childhood. About 300 people in the US and 900 worldwide, according to Mirum, have the condition.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">Mirum expects to launch the drug in October and plans to announce its price at the launch, chief executive officer Chris Peetz told Reuters.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">\u201cIt is a game changer for patients to have a medicine like this that can really stop that progressive, endless accumulation of bone,\u201d Peetz said.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">Citizens analyst Jonathan Wolleben estimates Atebrioz could cost about US$750,000 (S$958,410) annually and generate roughly US$150 million in peak worldwide sales.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">Mirum had licensed the drug from Incyte earlier in 2026 for US$16 million upfront, along with potential milestone payments and sales royalties.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">The approval by the US Food and Drug Administration was based on a 63-patient study, in which Atebrioz significantly cut new bone formation compared to placebo at week 24.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">Current treatment options for FOP include Ipsen\u2019s oral drug Sohonos and Regeneron\u2019s Pasatru, a monthly infusion. Sohonos is approved for certain children and adults, while Pasatru is approved for adults.<\/p>\n<p class=\"text-primary font-tertiary-body-baseline-regular\" data-testid=\"article-paragraph-annotation-test-id\">Wolleben said Atebrioz could become a preferred treatment because it combines oral dosing with strong efficacy and a favorable safety profile, offering greater convenience than Regeneron\u2019s monthly infusion and potentially fewer safety concerns than Ipsen\u2019s Sohonos. REUTERS<\/p>\n","protected":false},"excerpt":{"rendered":"WASHINGTON &#8211; The US FDA on Sept 25 approved Mirum Pharmaceuticals\u2019 pill for a rare bone disorder, offering&hellip;\n","protected":false},"author":2,"featured_media":908292,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[33],"tags":[64,63,137,490],"class_list":["post-908291","post","type-post","status-publish","format-standard","has-post-thumbnail","category-medication","tag-au","tag-australia","tag-health","tag-medication"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts\/908291","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/comments?post=908291"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts\/908291\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/media\/908292"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/media?parent=908291"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/categories?post=908291"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/tags?post=908291"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}