智通财经APP获悉，罗氏(RHHBY.US)（Roche）旗下基因泰克（Genentech）公司今日宣布，美国FDA已批准Evrysdi（risdiplam）片剂的新药申请（NDA），用于治疗脊髓性肌萎缩症（SMA）患者。新闻稿指出，Evrysdi是首款改变SMA疾病进程的非侵入性治疗药物。Evrysdi（5 mg）片剂既可整片吞服，也可溶解于水中服用。新闻稿指出，这是首款治疗SMA的片剂药物，其便捷的用药方式给SMA患者和护理者提供更多自由和独立性。
Evrysdi片剂的批准基于一项生物等效性研究结果，该研究表明，无论Evrysdi（5 mg）片剂是整片吞服还是溶解于非氯化饮用水（例如过滤水）中服用，与原有口服液相比，risdiplam在体内的暴露水平相当。这意味着服用该片剂的患者可获得与Evrysdi口服液相同的既定疗效和安全性。对于使用其他剂量Evrysdi以及可能更倾向于口服液的患者，Evrysdi口服液仍将保持供应。Evrysdi通过调节SMN2基因mRNA的剪接，提高能够表达正常SMN蛋白的mRNA水平，从而缓解SMA患者的症状。
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