华盛顿 - 美国食品药品监督管理局（FDA）于9月25日批准了Mirum Pharmaceuticals公司治疗一种罕见骨骼疾病的药丸，为患者提供了一种新的治疗选择，有助于减缓疾病进展。
WASHINGTON - The US FDA on Sept 25 approved Mirum Pharmaceuticals’ pill for a rare bone disorder, offering patients a new treatment option that can help slow the disease’s progression.
这种每日服用一次的药物zilurgisertib，商品名为Atebrioz，获批用于12岁及以上的进行性骨化性纤维发育不良（FOP）患者，该病会导致肌肉、肌腱和韧带逐渐转化为骨骼，限制活动并导致严重残疾。
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.
Atebrioz可阻断ALK2，这是一种在大多数FOP患者体内异常活跃并驱动骨骼外成骨的蛋白质。推荐剂量为100毫克。
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.
FOP通常在儿童早期确诊。据Mirum称，美国约有300人、全球约有900人患有此病。
FOP is typically diagnosed in early childhood. About 300 people in the US and 900 worldwide, according to Mirum, have the condition.
首席执行官Chris Peetz告诉路透社，Mirum预计将于10月推出该药物，并计划在发布时公布价格。
Mirum expects to launch the drug in October and plans to announce its price at the launch, chief executive officer Chris Peetz told Reuters.
Peetz表示：“拥有这样一种能够真正阻止骨骼进行性、无休止积累的药物，对患者来说是一个游戏规则的改变者。”
“It is a game changer for patients to have a medicine like this that can really stop that progressive, endless accumulation of bone,” Peetz said.
Citizens分析师Jonathan Wolleben估计，Atebrioz的年费用可能约为75万美元（约95.84万新元），并在巅峰期实现约1.5亿美元的全球销售额。
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.
Mirum于2026年早些时候从Incyte获得该药物许可，预付1600万美元，外加潜在的里程碑付款和销售特许权使用费。
Mirum had licensed the drug from Incyte earlier in 2026 for US$16 million upfront, along with potential milestone payments and sales royalties.
美国食品药品监督管理局的批准基于一项涉及63名患者的研究，结果显示，与安慰剂相比，Atebrioz在第24周显著减少了新骨形成。
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.
FOP目前的治疗选择包括Ipsen的口服药物Sohonos和Regeneron的Pasatru（一种每月输注疗法）。Sohonos获批用于特定儿童和成人，而Pasatru获批用于成人。
Current treatment options for FOP include Ipsen’s oral drug Sohonos and Regeneron’s Pasatru, a monthly infusion. Sohonos is approved for certain children and adults, while Pasatru is approved for adults.
Wolleben表示，Atebrioz有望成为一种首选疗法，因为它结合了口服给药方式、强大的疗效以及良好的安全性，与再生元（Regeneron）每月一次的输注疗法相比更为便捷，且相较于益普生（Ipsen）的Sohonos可能具有更少的安全性顾虑。
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’s monthly infusion and potentially fewer safety concerns than Ipsen’s Sohonos.