Santhera Pharmaceuticals has announced that the Chinese National Medical Products Administration (NMPA) has approved AGAMREE® (vamorolone) for patients aged 4 years and older in China. AGAMREE is a groundbreaking therapy with a unique mechanism of action. It binds to the same receptor as glucocorticoids but modifies the receptor's downstream activity, distinguishing itself from traditional corticosteroids. This distinctive mechanism enables AGAMREE to deliver therapeutic benefits while reducing safety concerns typically associated with steroids, positioning it as a dissociative anti-inflammatory and a novel alternative to standard corticosteroids for children and adolescents with Duchenne muscular dystrophy (DMD).In the pivotal VISION-DMD study, AGAMREE achieved its primary endpoint by significantly improving the standing-to-stand speed (TTSTAND) compared to placebo (p=0.002) after 24 weeks of treatment.

Santhera Pharmaceuticals宣布，中国国家医药产品管理局（NMPA）已批准AGAMREE®（vamorolone）用于中国4岁及以上的患者。AGAMREE是一种具有独特作用机制的开创性疗法。它与糖皮质激素的受体结合，但会改变受体的下游活性，从而与传统的皮质类固醇区别开来。。在关键的VISION-DMD研究中，治疗24周后，与安慰剂（p=0.002）相比，AGAMREE通过显着提高站立速度（TTSTAND）来实现其主要终点。

It also demonstrated a favourable safety and tolerability profile, with the most common adverse events being Cushingoid appearance, vomiting, weight gain, and irritability—generally mild to moderate in severity.AGAMREE marks a milestone as the first and only approved treatment for DMD in China. This approval follows the acceptance of the New Drug Application (NDA) by the NMPA in March 2024.

它还表现出良好的安全性和耐受性，最常见的不良事件是库欣样外观，呕吐，体重增加和烦躁不安，严重程度一般为轻度至中度。AGAMREE标志着一个里程碑，它是中国第一个也是唯一一个获得批准的DMD治疗方法。该批准是在2024年3月NMPA接受新药申请（NDA）之后获得的。

The NDA highlighted AGAMREE’s clinically significant safety benefits, including the maintenance of normal bone metabolism, density, and growth, compared to traditional corticosteroids. DMD is a debilitating condition that affects over 70,000 individuals in China. Until now, there were no approved treatment options for this patient population, making AGAMREE's approval a significant advancement in addressing this critical unmet need. Source: globenewswire.com.

NDA强调了与传统皮质类固醇相比，AGAMREE的临床显着安全益处，包括维持正常的骨代谢，密度和生长。DMD是一种使人衰弱的疾病，影响着中国70000多人。到目前为止，还没有针对该患者群体的批准治疗方案，这使得阿加姆雷的批准在解决这一关键的未满足需求方面取得了重大进展。来源：globenewswire.com。