)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi

)--罗氏集团（SIX:RO，ROG；OTCQX:RHHBY）的成员基因泰克（Genentech）今天宣布，美国食品和药物管理局（FDA）已批准Evrysdi的新药申请（NDA）

(risdiplam) tablet for people living with spinal muscular atrophy (SMA). Evrysdi is the only non-invasive disease-modifying treatment for SMA. The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water.

（利培林）片剂，用于脊髓性肌萎缩症（SMA）患者。Evrysdi是SMA唯一的非侵入性疾病缓解治疗方法。5毫克Evrysdi片剂可以全部吞咽或分散在水中。

“Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.”.

“Evrysdi具有改变SMA疾病轨迹的强大潜力，迄今已被用于治疗数千名患者。这一批准标志着又向前迈出了重要的一步，”基因泰克首席医疗官兼全球产品开发负责人Levi Garraway博士说。“Evrysdi平板电脑将既定的功效与便利性相结合，为SMA管理提供了额外的灵活选择。”。

The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking water (e.g., filtered water), and original oral solution provide comparable exposure to risdiplam. This means patients who take the tablet can expect the same established efficacy and safety as the Evrysdi oral solution.

Evrysdi片剂的批准是基于生物等效性研究的结果，该研究表明，5毫克片剂无论是全部吞咽还是分散在非氯化饮用水（例如过滤水）和原始口服溶液中，都可以提供与利培林相当的暴露量。这意味着服用该片剂的患者可以预期与Evrysdi口服溶液相同的既定疗效和安全性。

The Evrysdi oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution..

Evrysdi口服溶液将继续适用于其他剂量的Evrysdi患者和可能更喜欢口服溶液的患者。。

“We cannot underestimate the value that comes with simplifying treatment administration and disease management for people who are living with SMA or those caring for them,” said Kenneth Hobby, President of Cure SMA. “This new room temperature stable formulation option offers an additional choice that may more conveniently fit into daily living activities such as working, traveling, and education.”.

Cure SMA总裁肯尼斯·哈比（KennethHobby）说：“我们不能低估简化治疗管理和疾病管理对SMA患者或照顾他们的人的价值。”。“这种新的室温稳定配方选择提供了一种额外的选择，可以更方便地适应日常生活活动，如工作，旅行和教育。”。

As part of the label extension, the Evrysdi prescribing information has been updated to include guidance on tablet administration and storage.

作为标签扩展的一部分，Evrysdi处方信息已经更新，包括关于平板电脑管理和存储的指导。

The new tablet, expected to be available in the coming weeks, is suitable for people 2 years of age or older who weigh more than 44 lbs (20 kgs).

这款新平板电脑预计将于未来几周上市，适合2岁或2岁以上体重超过44磅（20公斤）的人群。

Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

作为与SMA基金会和PTC Therapeutics合作的一部分，Genentech领导Evrysdi的临床开发。

About Evrysdi®

关于Evrysdi®

Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home or on the go, either in liquid form (by feeding tube or by mouth) or in the form of a tablet (by mouth only, swallowed whole or dispersed in water)..

Evrysdi是一种存活的运动神经元2(SMN2)前mRNA剪接修饰剂旨在治疗由5q染色体突变引起的SMA，该突变导致运动神经元（SMN）蛋白缺乏症的存活。Evrysdi每天在家或外出时服用，无论是液体形式（通过饲管或口服）还是片剂形式（仅通过口服，全部吞咽或分散在水中）。。

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS) and peripheral tissues, as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core functions..

Evrysdi旨在通过增加和维持中枢神经系统（CNS）和外周组织中SMN蛋白的产生来治疗SMA，如动物模型所示。SMN蛋白遍布全身，对维持健康的运动神经元和核心功能至关重要。。

Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research Award for Drug Discovery.

Evrysdi于2018年被欧洲药品管理局（EMA）授予最佳称号，2017年被美国食品和药物管理局（FDA）授予孤儿药称号。2021年，Evrysdi被英国药理学学会授予年度药物发现奖，以及药物研究学会授予药物发现奖。

Evrysdi is currently approved in more than 100 countries, with more than 16,000 people with SMA treated globally..

Evrysdi目前已在100多个国家获得批准，全球有16000多名SMA患者接受治疗。。

Evrysdi is currently being, or has been, evaluated in numerous global multicenter trials in people with SMA:

Evrysdi目前正在或已经在许多针对SMA患者的全球多中心试验中进行评估：

FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately 5.5 months of age (median) at the time of enrollment and of the 58 infants that completed the first year of treatment, 52 entered the open-label extension study. The study met its primary endpoint and has concluded after 5 years of follow up..

FIREFISH（NCT02913482）–一项针对1型SMA婴儿的开放标签、两部分关键临床试验。入组时婴儿约为5.5个月大（中位数），在完成第一年治疗的58名婴儿中，有52名进入了开放标签扩展研究。该研究达到了其主要终点，并在5年的随访后得出结论。。

SUNFISH (NCT02908685) – a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. The study met its primary endpoint and has concluded after 5 years of follow up.

SUNFISH（NCT02908685）–一项针对2-25岁2型或3型SMA患者的两部分、双盲、安慰剂对照关键研究。该研究达到了其主要终点，并在5年的随访后得出结论。

JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies prior to receiving Evrysdi. The study has completed recruitment (n=174)..

JEWELFISH（NCT03032172）-一项开放标签探索性试验，旨在评估6个月至60岁的SMA患者的安全性，耐受性，药代动力学和药效学，这些患者在接受Evrysdi之前接受了其他研究或批准的SMA治疗。该研究已完成招聘（n=174）。。

RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies (n=26), from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study met its primary endpoint..

RAINBOWFISH（NCT03779334）-一项开放标签，单臂，多中心研究，调查Evrysdi在婴儿（n=26）中的有效性，安全性，药代动力学和药效学，从出生到6周龄（第一次服用），遗传诊断的SMA尚未出现症状。该研究达到了其主要终点。。

MANATEE (NCT05115110) – a Phase II/III clinical study to evaluate the safety and efficacy of GYM329 (RG6237), an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2 to 10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021.

MANATEE（NCT05115110）–一项II/III期临床研究，旨在评估针对肌肉生长的抗肌肉生长抑制素分子GYM329（RG6237）与Evrysdi联合治疗2至10岁患者SMA的安全性和有效性。2021年12月，FDA孤儿产品开发办公室批准GYM329孤儿药用于治疗SMA患者。

The study is currently active..

该研究目前正在进行中。。

HINALEA 1 (NCT05861986) and HINALEA 2 (NCT05861999) – Phase IV clinical studies to evaluate the effectiveness and safety of Evrysdi in patients under 2 years of age at enrollment, who received onasemnogene abeparvovec gene therapy either pre-symptomatically or post-symptomatically, following a genetically confirmed diagnosis of 5q–autosomal recessive SMA.

HINALEA 1（NCT05861986）和 HINALEA 2（NCT05861999）--IV 期临床研究，旨在评估 Evrysdi 在入组时未满 2 岁的患者中的有效性和安全性，这些患者在症状出现前或症状出现后接受了 onasemnogene abeparvovec 基因疗法，并经基因确诊为 5q 常染色体隐性 SMA。

PUPFISH (NCT05808764) – a Phase II, open-label study to investigate the pharmacokinetics and safety of Evrysdi in babies with SMA who are under 20 days of age (at first dose). The study is currently recruiting.

PUPFISH（NCT05808764）–一项II期开放标签研究，旨在研究Evrysdi在20日龄以下（首次给药）SMA婴儿中的药代动力学和安全性。这项研究目前正在招募。

About SMA

关于SMA

SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is among the leading genetic causes of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (

SMA是一种严重的进行性神经肌肉疾病，可能致命。它影响大约10000名婴儿中的一名，是导致婴儿死亡的主要遗传原因之一。SMA是由存活运动神经元1的突变引起的(

SMN1

SMN1

) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost..

)基因，导致SMN蛋白缺乏。这种蛋白质遍布全身，对控制肌肉和运动的神经功能至关重要。没有它，神经细胞就无法正常工作，随着时间的推移会导致肌肉无力。根据SMA的类型，个人的体力和行走、进食或呼吸的能力可能会显着降低或丧失。。

What is Evrysdi?

什么是Evrysdi？

Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

Evrysdi是一种用于治疗儿童和成人脊髓性肌萎缩症（SMA）的处方药。