Roche has received approval from the European Commission for a new 5mg tablet formulation of Evrysdi® (risdiplam), the first and only non-invasive, disease-modifying treatment for spinal muscular atrophy (SMA).

罗氏公司已获得欧洲委员会批准，推出Evrysdi®（risdiplam）新的5毫克片剂配方，这是首个也是唯一一个用于治疗脊髓性肌萎缩症（SMA）的非侵入性、疾病修饰疗法。

Spinal muscular atrophy is a rare and progressive neuromuscular disorder caused by a mutation in the SMN1 gene, leading to a deficiency in SMN protein. This deficiency disrupts the functioning of nerves that control muscle movement, potentially affecting the ability to walk, eat, or breathe. SMA affects roughly one in 10,000 newborns and remains a leading genetic cause of infant death..

脊髓性肌萎缩症是一种罕见的、进行性的神经肌肉疾病，由SMN1基因突变引起，导致SMN蛋白缺乏。这种缺乏会破坏控制肌肉运动的神经功能，可能影响行走、进食或呼吸的能力。SMA大约每10,000名新生儿中就有一例，仍是婴儿死亡的主要遗传原因之一。

The tablet is stable at room temperature, can be swallowed whole or mixed with water, and does not require refrigeration. It offers greater flexibility and convenience for people living with SMA, supporting at-home administration and easing daily treatment routines.

片剂在室温下稳定，可整片吞服或与水混合服用，无需冷藏。它为SMA患者提供了更大的灵活性和便利性，支持在家用药，简化了每日的治疗流程。

Evrysdi remains the only non-invasive, disease-modifying treatment available for SMA and has already been used to treat over 18,000 people globally. This new formulation further enhances ease of administration, particularly for those aged two years and above who weigh at least 20kg and can swallow tablets without a feeding tube..

Evrysdi仍然是唯一可用的非侵入性、改变疾病的SMA治疗方法，并且已经在全球范围内用于治疗超过18,000人。这种新配方进一步简化了给药方式，特别是适用于两岁及以上、体重至少20公斤且无需喂食管即可吞咽药片的患者。

The tablet formulation demonstrated bioequivalence with the original oral solution, as confirmed by data from a clinical study (NCT04718181) presented at SMA Europe’s Scientific International Congress in 2024. This means it offers the same safety and effectiveness as the liquid version. The original oral solution remains available for those on different doses or who prefer it..

片剂配方在一项临床研究（NCT04718181）中显示出与原口服溶液的生物等效性，该数据在2024年SMA欧洲科学国际大会上公布。这意味着它与液体版本具有相同的安全性和有效性。原先的口服溶液仍然可供那些使用不同剂量或更喜欢它的人使用。

Evrysdi works by boosting and maintaining the production of SMN protein in both the central nervous system and peripheral tissues. This protein is essential for motor neuron health and overall muscle function.

Evrysdi通过提升并维持中枢神经系统和周围组织中SMN蛋白的产生来发挥作用。这种蛋白质对于运动神经元健康和整体肌肉功能至关重要。

Roche continues to lead the development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics.

罗氏继续与SMA基金会和PTC Therapeutics合作，领导Evrysdi的开发。

The treatment has received numerous regulatory designations, including PRIME status from the European Medicines Agency and Orphan Drug Designation from the U.S. FDA.

该治疗方案已获得多项监管指定，包括欧洲药品管理局的优先药物（PRIME）资格和美国FDA的孤儿药认定。