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The US FDA on Friday approved \u200bMirum Pharmaceuticals\u2019 pill for a rare bone disorder, offering patients a \u200cnew treatment option that can help slow the disease\u2019s progression.<\/strong><\/p>\n The once-daily drug zilurgisertib, branded as Atebrioz, is approved for patients aged 12 years and older with fibrodysplasia ossificans progressiva (FOP), which \u200bcauses muscles, tendons and ligaments to gradually transform into bone, restricting movement \u200band leading to severe disability.<\/p>\n Atebrioz blocks ALK2, a protein that is \u2060abnormally active in most patients with FOP and drives bone formation outside \u200bthe skeleton. The recommended dose is 100 milligrams.<\/p>\n FOP is typically diagnosed in early childhood. \u200bAbout 300 people in the US and 900 worldwide, according to Mirum, have the condition.<\/p>\n Mirum expects to launch the drug in October and plans to announce its price at the launch, \u200bCEO Chris Peetz told Reuters.<\/p>\n \u201cIt is a game-changer for patients to have a \u200bmedicine like this that can really stop that progressive, endless accumulation of bone,\u201d Peetz said.<\/p>\n Citizens \u200canalyst \u2060Jonathan Wolleben estimates Atebrioz could cost about $750,000 annually and generate roughly $150 million in peak worldwide sales.<\/p>\n Mirum had licensed the drug from Incyte earlier this year for $16 million upfront, along with potential milestone payments and sales royalties.<\/p>\n The approval by the US Food \u200band Drug Administration \u200bwas based on \u2060a 63-patient study, in which Atebrioz significantly cut new bone formation compared to placebo at week 24.<\/p>\n Current treatment options for \u200bFOP include Ipsen\u2019s oral drug Sohonos and Regeneron\u2019s Pasatru, a \u200bmonthly infusion. \u2060Sohonos is approved for certain children and adults, while Pasatru is approved for adults.<\/p>\n Wolleben said Atebrioz could become a preferred treatment because it combines oral dosing with strong \u2060efficacy \u200band a favorable safety profile, offering greater convenience \u200bthan Regeneron\u2019s monthly infusion and potentially fewer safety concerns than Ipsen\u2019s Sohonos.<\/p>\n<\/div>\n