STOCKHOLM, Sept. 27, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ('Calliditas') today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Alport syndrome with setanaxib.

斯德哥尔摩，2023年9月27日/PRNewswire/-Calliditas Therapeutics AB（纳斯达克股票代码：CALT）（纳斯达克股票代码：CALTX）（'Calliditas'）今天宣布美国食品和药物管理局（FDA）已授予孤儿药名称（ODD）给公司用司他昔布治疗Alport综合征。

Based on supportive pre-clinical work, Calliditas plans to initiate a randomized, placebo-controlled phase 2 clinical study in Alport syndrome with around 20 patients in the fourth quarter of 2023.

基于支持性的临床前工作，Calliditas计划在2023年第四季度启动Alport综合征的随机，安慰剂对照2期临床研究，约20名患者。

'We are excited to start another clinical program in the renal space targeting an orphan indication where today there are no approved products,' said CEO Renée Aguiar-Lucander.

首席执行官RenéeAguiar-Lucander说：“我们很高兴在肾脏领域开始另一项针对孤儿适应症的临床项目，目前还没有获得批准的产品。

Alport syndrome is a genetic disorder arising from the mutations in the genes that code for type 4 collagen. The type 4 collagen alpha chains are primarily located in the kidneys, eyes, and cochlea, and thus the condition is characterized by kidney disease, loss of hearing, and eye abnormalities. Eventually, patients present with proteinuria, hypertension, progressive loss of kidney function (gradual decline in GFR), and end-stage renal disease (ESRD).

Alport综合征是一种遗传性疾病，由编码4型胶原蛋白的基因突变引起。4型胶原α链主要位于肾脏，眼睛和耳蜗中，因此该疾病的特征是肾脏疾病，听力下降和眼睛异常。最终，患者出现蛋白尿，高血压，肾功能进行性丧失（GFR逐渐下降）和终末期肾病（ESRD）。

It is estimated that approximately 30,000 to 60,000 people in the United States (US) have this disorder, and it is a significant cause of chronic kidney disease (CKD), leading to ESRD in adolescents and young adults and accounting for 1.5% to 3.0% of children on renal replacement therapies in Europe and the US.[1].

据估计，美国（美国）约有30000至60000人患有这种疾病，并且是慢性肾病（CKD）的重要原因，导致青少年和年轻人的ESRD，占1.5%至3.0%欧洲和美国的肾脏替代疗法儿童[1]。

Through its subsidiary Calliditas Therapeutics Suisse SA, which will sponsor the planned phase 2 clinical study of setanaxib in Alport syndrome, Calliditas is currently investigating setanaxib in a Phase 2 proof-of-concept study in squamous cell carcinoma of the head and neck (SCCHN), as well as in a Phase 2b study in primary biliary cholangitis (PBC).

通过其子公司Calliditas Therapeutics Suisse SA，该公司将赞助计划在Alport综合征中进行的setanaxib 2期临床研究，Calliditas目前正在头颈部鳞状细胞癌（SCCHN）的2期概念验证研究中研究setanaxib。），以及原发性胆汁性胆管炎（PBC）的2b期研究。

Setanaxib is also being evaluated in an investigator-led study in idiopathic pulmonary fibrosis (IPF)..

Setanaxib也正在一项研究者主导的特发性肺纤维化（IPF）研究中进行评估。。

[1] Watson S, Padala SA, Hashmi MF, et al. Alport Syndrome. [Updated 2023 Aug 14]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK470419/

[1] Watson S，Padala SA，Hashmi MF，et al.Alport Syndrome。[于2023年8月14日更新]。在：StatPearls[互联网]。宝岛（FL）：StatPearls出版社；2023年1月-。可从：https://www.ncbi.nlm.nih.gov/books/NBK470419/

For further information, please contact:

有关详细信息，请联系：

Åsa Hillsten, Head of IR & Sustainability, CalliditasTel.: +46 76 403 35 43, Email: [email protected]

Åsa Hillsten，CalliditasTel红外与可持续发展负责人：+46 76 403 35 43，电子邮件：[电子邮件保护]

The following files are available for download:

以下文件可供下载：

https://mb.cision.com/Main/16574/3843536/2324182.pdf

https://mb.cision.com/Main/16574/3843536/2324182.pdf

CALSE Alport ODD PR (2023.09.27)

CALSE Alport ODD PR（2023.09.27）

SOURCE Calliditas Therapeutics

源Callidites疗法