LUND, Sweden, Feb. 05, 2024 (GLOBE NEWSWIRE) -- AnaMar, a clinical-stage biotech company developing first-in-class anti-fibrotic 5-HT2B receptor antagonists, announces that the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have granted orphan drug designation (ODD) to its lead clinical candidate, AM1476 for the treatment of systemic sclerosis (SSc).

瑞典隆德，2024年2月5日（环球通讯社）--开发一流抗纤维化5-HT2B受体拮抗剂的临床阶段生物技术公司AnaMar宣布，欧洲药品管理局（EMA）和美国食品和药物管理局（FDA）已将孤儿药名称（ODD）授予其主要临床候选药物AM1476，用于治疗系统性硬化症（SSc）。

AM1476 is a selective peripheral-acting 5-HT2B receptor small molecule antagonist that can be delivered orally. It has demonstrated efficacy in in vitro and in vivo models of fibrosis, as well as favourable safety and pharmacokinetic profiles in Phase I clinical studies. AM1476 offers a unique dual-action approach to treat both skin and lung manifestations of systemic sclerosis.

AM1476是一种选择性外周作用的5-HT2B受体小分子拮抗剂，可口服。它已经证明在纤维化的体外和体内模型中有效，以及在I期临床研究中有利的安全性和药代动力学特征。AM1476提供了一种独特的双重作用方法来治疗系统性硬化症的皮肤和肺部表现。

Systemic sclerosis is a chronic, progressive, autoimmune disease characterized by inflammation and fibrosis, i.e. uncontrolled scar tissue formation, in skin and various internal organs. Skin fibrosis is the distinguishing hallmark of SSc, associated with significant disability. The degree of skin fibrosis provides insight about progression of the disease.

系统性硬化症是一种慢性，进行性，自身免疫性疾病，其特征在于皮肤和各种内脏器官中的炎症和纤维化，即不受控制的瘢痕组织形成。皮肤纤维化是SSc的显着标志，与严重残疾有关。皮肤纤维化程度提供了有关疾病进展的见解。

Interstitial lung disease (ILD) is a common and early manifestation of SSc. Around 100,000 people in the EU are affected by SSc and up to 80% of these may develop ILD. ILD causes progressive lung scarring, known as fibrosis, which leads to increasing, chronic, breathing problems and organ dysfunction, and can lead to death.

间质性肺病（ILD）是SSc的常见早期表现。欧盟约有10万人受到SSc的影响，其中高达80%的人可能会患ILD。ILD导致进行性肺瘢痕形成，称为纤维化，导致慢性呼吸问题和器官功能障碍增加，并可能导致死亡。

There are currently no treatments on the market that effectively stop or reverse scarring in both skin and lung tissue. AnaMar's Chief Executive Officer, Dr. Ulf Ljungberg, said: “We are delighted with the FDA’s and EMA’s decisions to grant orphan drug designation to AM1476 for SSc. This is a significant milestone and underscores the .

目前市场上还没有有效阻止或逆转皮肤和肺组织瘢痕形成的治疗方法。AnaMar的首席执行官Ulf Ljungberg博士说：“我们很高兴FDA和EMA决定将孤儿药指定为AM1476用于SSc。这是一个重要的里程碑，并强调了这一点。