PTC Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU..

PTC Therapeutics今天宣布，美国食品和药物管理局 (FDA) 已批准 SEPHIENCE™（sepiapterin）用于治疗苯丙酮尿症 (PKU) 的儿童和成人患者。该批准包括针对 1 个月及以上年龄的成人和儿童患者的高苯丙氨酸血症 (HPA) 治疗的广泛标签，适用于对 sepiapterin 有反应的 PKU 患者。

'We are excited to have reached this important milestone for those affected by PKU,' said

“我们很高兴能为那些受PKU影响的人达成这一重要里程碑，”

Matthew B. Klein

马修·B·克莱因

, M.D., Chief Executive Officer of PTC Therapeutics. 'The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients. The Sephience clinical data along with our expertise in launching rare disease therapies position Sephience to become the future standard of care.

医学博士，PTC Therapeutics首席执行官表示：“广泛的标签反映了Sephience满足苯丙酮尿症（PKU）患者重大未满足需求的潜力。Sephience的临床数据以及我们在推出罕见病治疗方面的专业能力，使Sephience有望成为未来的护理标准。”

Our experienced customer facing teams are ready to bring this therapy to children and adults with PKU in .

我们经验丰富的面向客户的团队已准备好将这种疗法带给苯丙酮尿症（PKU）的儿童和成人。

the United States

美国

as quickly as possible.'

尽可能快地。

The FDA approval is based on the evidence of significant efficacy and safety from the

FDA 的批准是基于显著的有效性和安全性的证据

Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study.

第三阶段APHENITY试验以及APHENITY长期扩展研究中的治疗效果持久性。

'The approval marks an exciting milestone for the PKU community,' said

“这一批准标志着PKU社区令人兴奋的里程碑，”

Catherine Warren

凯瑟琳·沃伦

, Executive Director of the National PKU Alliance. 'This progress brings renewed hope, and we are eager to see the positive impact this new treatment option will have on advancing care and potentially improving quality of life for individuals of all ages and PKU subtypes that respond to this therapy.'.

，国家PKU联盟执行董事。“这一进展带来了新的希望，我们渴望看到这种新的治疗选择将对提升护理水平和潜在改善所有年龄和PKU亚型患者的生活质量产生积极影响。”

SEPHIENCE was recently granted marketing authorization by the European Commission. Review of approval applications is ongoing in several other countries including

SEPHIENCE最近获得了欧盟委员会的上市授权。包括其他国家在内的几个国家正在对批准申请进行审查。

Japan

日本

and

和

Brazil

巴西

About SEPHIENCE™ (sepiapterin)

关于SEPHIENCE™（sepiapterin）

SEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients.

SEPHIENCE 适用于治疗成年和儿童苯丙酮尿症 (PKU) 患者的高苯丙氨酸血症 (HPA)。Sephience 是酶辅因子 BH4 的天然前体，BH4 是苯丙氨酸羟化酶 (PAH) 的关键辅因子。通过其作用机制，Sephience 能够有效降低血液中的苯丙氨酸 (Phe) 水平，并有潜力治疗广泛范围的 PKU 患者。

Sephience is approved in the European Economic Area and .

Sephience 已获欧洲经济区批准。

About Phenylketonuria

关于苯丙酮尿症

Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine (Phe). If left untreated or poorly managed, Phe—an essential amino acid found in all proteins and most foods—can build up to harmful levels in the body.

苯丙酮尿症（PKU）是一种罕见的遗传性代谢疾病，会影响大脑。它是由帮助生成分解苯丙氨酸（Phe）所需酶的基因缺陷引起的。如果不治疗或管理不当，Phe（一种存在于所有蛋白质和大多数食物中的必需氨基酸）可能在体内积累到有害水平。

This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with PKU initially do not have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible.

这会导致严重且不可逆的残疾，如永久性智力障碍、癫痫发作、发育迟缓、记忆力丧失以及行为和情绪问题。患有PKU的新生儿最初没有任何症状，但症状通常会逐渐加重，生命头几年因苯丙氨酸毒性水平造成的损害是不可逆的。

Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU globally..

苯丙酮尿症的诊断通常在新生儿筛查项目中进行。全球约有58,000人患有苯丙酮尿症。

About PTC Therapeutics, Inc.

关于PTC治疗公司

PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs.

PTC是一家全球生物制药公司，致力于为患有罕见疾病的儿童和成人发现、开发和商业化临床差异化的药物。PTC正在推进一系列强大且多样化的变革性药物管线，作为其为未满足医疗需求的患者提供最佳治疗方案使命的一部分。

The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit .

公司的战略是利用其科学专业知识和全球商业基础设施，为患者和其他利益相关者优化价值。要了解更多信息，请访问PTC官网。