/PRNewswire/ -- Vascarta Inc., a biopharmaceutical company committed to advancing innovative therapies for underserved patient populations, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead drug candidate, Vasceptor

/PRNewswire/ -- 致力于为服务不足的患者群体推进创新疗法的生物制药公司 Vascarta Inc. 今天宣布，美国食品和药物管理局 (FDA) 已授予其主要候选药物 Vasceptor 孤儿药资格。

(VAS-101), for the treatment of Sickle Cell Disease (SCD).

(VAS-101)，用于治疗镰状细胞病 (SCD)。

Orphan Drug Designation is awarded to therapies intended to treat rare diseases that affect fewer than 200,000 people in

获得孤儿药资格的疗法旨在治疗影响美国不到20万人的罕见疾病，

the United States

美国

. This designation provides significant benefits, including seven years of market exclusivity upon approval, tax credits for qualified clinical trials, and exemption from certain FDA fees.

这一指定提供了显著的好处，包括在获批后七年的市场独占期、符合条件的临床试验的税收抵免，以及豁免某些FDA费用。

'The attainment of Orphan Drug Designation represents an important regulatory milestone for Vascarta and underscores the therapeutic potential of Vasceptor

“获得孤儿药资格认证代表了Vascarta的一个重要监管里程碑，并突显了Vasceptor的治疗潜力。

to provide safe and effective relief to patients living with this debilitating disease,' said Dr.

为患有这种衰弱性疾病的患者提供安全有效的缓解，"博士说。

Richard Prince

理查德·普林斯

, CEO & President of Vascarta. 'We plan to work closely with the United States Food & Drug Administration to bring Vasceptor

，Vascarta的首席执行官兼总裁。“我们计划与美国食品药品监督管理局密切合作，推出Vasceptor

to market as rapidly as possible.'

尽快上市。

Dr. Joel Friedman, Professor, Department of Microbiology & Immunology, Albert Einstein College of Medicine, and Vascarta Scientific Founder & Head, Scientific Advisory Board, stated, 'The preclinical results to date showing pain reduction and therapeutic efficacy resulting from our novel approach of targeting red blood cell instability, neuro-inflammation and vascular inflammation bodes well for the development of a widely accessible therapy that prevents and treats many if not most of the clinical consequences of SCD.' Vasceptor.

阿尔伯特·爱因斯坦医学院微生物学与免疫学系教授、Vascarta科学创始人兼科学顾问委员会主席乔尔·弗里德曼博士表示：“迄今为止的临床前结果表明，我们针对红细胞不稳定、神经炎症和血管炎症的新方法能够减轻疼痛并具有治疗效果，这为开发一种广泛可用的疗法带来了良好的前景，这种疗法可以预防并治疗镰状细胞病（SCD）的许多甚至大部分临床后果。”

is exclusively licensed to Vascarta from the Albert Einstein College of Medicine (

由阿尔伯特·爱因斯坦医学院独家授权给Vascarta (

Bronx, New York

纽约州布朗克斯区

, USA).

，美国）。

About

关于

Vasceptor

血管钳

VAS-101)

VAS-101)

VAS-101, developed by Vascarta Inc. (

VAS-101，由Vascarta Inc.开发（

Summit, NJ

新泽西州萨米特

, USA), is a patented topical curcumin formulation that is delivered using patented transdermal technology and that is designed to overcome the limited bioavailability and suboptimal effectiveness of oral dosing of curcumin. A study in

，美国），是一种使用专利透皮技术递送的专利局部姜黄素制剂，旨在克服姜黄素口服给药生物利用度有限和效果欠佳的问题。一项研究显示

suggests VAS-101 may reduce chronic pain, stabilize red blood cells, and lower inflammation in sickle cell disease (SCD). It is currently in a Phase 1 clinical trial at the Foundation for Sickle Cell Disease Research (FSCDR) in

表明VAS-101可能减少慢性疼痛、稳定红细胞并降低镰状细胞病（SCD）中的炎症。它目前正在进行一项由镰状细胞病研究基金会（FSCDR）开展的1期临床试验。

Hollywood, Florida

佛罗里达州好莱坞

. While curcumin is known for its safety and anti-inflammatory, antioxidant, and anti-sickling properties, its clinical use has been limited by poor oral bioavailability.

虽然姜黄素因其安全性、抗炎、抗氧化和抗镰刀形红细胞特性而闻名，但其临床应用受到口服生物利用度低的限制。

About Sickle Cell Disease

关于镰状细胞病

Sickle Cell Disease is the most common inherited genetic disorder that affects primarily African American and non – Hispanic Black individuals in the

镰状细胞病是最常见的遗传性疾病，主要影响非洲裔美国人和非西班牙裔黑人个体。

United States

美国

. SCD associated complications include anemia, acute and chronic pain, infections, pneumonia and acute chest syndrome, stroke, kidney, liver, and heart disease.

镰状细胞病相关的并发症包括贫血、急性和慢性疼痛、感染、肺炎和急性胸综合征、中风、肾脏、肝脏和心脏疾病。

Current estimates indicate there are 175,000 cases of SCD in the USA and 45,000 in European Union countries. The estimated life expectancy of those with sickle cell disease in the USA is more than 20 years shorter than the average expected lifespan.

目前的估计显示，美国有175,000例镰状细胞病（SCD）患者，欧盟国家有45,000例。美国镰状细胞病患者的预期寿命比平均预期寿命短20多年。

SCD is caused by a single point mutation in the globin gene leading to sickling of red blood cells. It is characterized by severe pain, inflammation, oxidative stress, and organ damage, which contributes to the poor quality of life and reduced survival. Recently approved SCD therapies do not mitigate pain, and patients are often on multiple drugs which often have undesirable side effects. .

镰状细胞病是由珠蛋白基因的单点突变引起的，导致红细胞镰状化。其特征是严重疼痛、炎症、氧化应激和器官损伤，这些因素导致生活质量下降和生存期缩短。最近获批的镰状细胞病疗法并不能缓解疼痛，患者通常需要服用多种药物，而这些药物往往伴有不良副作用。

The unmet medical need for most SCD sufferers is significant. Better therapies that are safe, improve outcomes, optimize compliance for patients of all ages, minimize the need for blood transfusions, and reduce the need for chronic administration of potentially harmful pain medications are needed.

大多数SCD患者未满足的医疗需求是显著的。需要更安全、改善疗效、优化各年龄段患者依从性、尽量减少输血需求并降低长期使用潜在有害止痛药需求的更好疗法。

About Vascarta

关于Vascarta

Vascarta Inc. is a clinical stage pharmaceutical company developing efficient transdermal and transmucosal delivery of pharmaceuticals to address inflammatory conditions with an initial focus on Sickle Cell Disease and osteoarthritis. To learn more, contact Vascarta Chairman, CEO & President, Dr. Richard Prince, at .

Vascarta Inc. 是一家临床阶段的制药公司，致力于开发高效的透皮和透黏膜药物递送系统，以应对炎症性疾病，最初专注于镰状细胞病和骨关节炎。欲了解更多信息，请联系 Vascarta 董事长、首席执行官兼总裁 Richard Prince 博士。