生产问题阻碍 Scholar Rock SMA药物获得 FDA批准Manufacturing issues block FDA nod for Scholar Rock SMA drug

动脉网2025-09-23

Unresolved compliance issues at a manufacturing plant operated by Novo Nordisk have led to a complete response letter for Scholar Rock's lead drug for spinal muscular atrophy (SMA).诺和诺德运营的一家制造工厂存在未解决的...

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In a statement, the Cambridge, Massachusetts-based company said that the CRL included no observations that were specific to its drug, such as safety, efficacy, or drug substance manufacturing.在一份声明中，这家位于马萨诸塞州剑桥的公司表示，完整回应函（CRL）中没有任何针对其药物的具体意见，如安全性、有效性或药物成分生产等方面的问题。'We are continuing to work closely with Catalent Indiana on the FDA's manufacturing observations so that we can resubmit the apitegromab BLA as soon as possible,' said David Hallal, Scholar Rock's chairman and chief executive.“我们正继续与Catalent印第安纳紧密合作，处理FDA对生产情况的观察意见，以便我们能尽快重新提交apitegromab的生物制品许可申请（BLA），” Scholar Rock董事长兼首席执行官David Hallal表示。'We remain focused on working hand-in-hand with the FDA to pursue approval of the first and only muscle-targeted treatment for people living with SMA,' he added.“我们仍然专注于与FDA携手合作，寻求批准首个也是唯一一个针对肌肉的SMA患者治疗方法，”他补充道。SMA脊髓性肌萎缩症is a genetic disorder that can cause rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing, and basic movement.是一种遗传性疾病，可导致运动神经元迅速且不可逆的丧失，影响包括呼吸、吞咽和基本运动在内的肌肉功能。Scholar Rock filed for approval of apitegromab based on the SAPPHIRE trial, which tested the drug in patients aged two to 12 who were already being treated with the standard therapies for SMA, Biogen's antisense-based therapy Spinraza (nusinersen) and Roche's oral mRNA splicing modifier Evrysdi (risdiplam), which prevent motor neuron loss but do not directly address muscle..Scholar Rock 基于 SAPPHIRE 试验申请了 apitegromab 的批准，该试验在已经接受 SMA 标准治疗（Biogen 的反义疗法 Spinraza（nusinersen）和 Roche 的口服 mRNA 剪接修饰剂 Evrysdi（risdiplam））的 2 至 12 岁患者中测试了该药物，这些疗法可防止运动神经元损失，但并未直接针对肌肉。It achieved a significant improvement in motor function over placebo after 52 weeks of treatment, measured using the 33-point Hammersmith Functional Motor Scale Expanded (HFMSE), which measures a range of physical activities.在使用33分的哈默史密斯功能运动量表扩展版（HFMSE）进行测量时，该量表评估了一系列身体活动，经过52周的治疗后，其运动功能相较于安慰剂组有了显著改善。Shares in Scholar Rock lost almost 13% of their value on the Nasdaq after news of the CRL emerged, although the stock has generally been weakening since news of the problems at the Catalent plant were first disclosed.纳斯达克市场上 Scholar Rock 的股价在 CRL 消息传出后下跌了近 13%，不过自 Catalent 工厂问题首次披露以来，该股总体上一直在走弱。Outside the US, apitegromab is under review by the EMA, with a decision expected in the middle of next year. 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