NEW TAIPEI CITY, March 3, 2024 /PRNewswire/ -- Caliway Biopharmaceuticals (Caliway) announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to CBL-514 for Dercum's disease treatment. CBL-514 is a lipolysis injection that can reduce local subcutaneous fat by inducing adipocyte apoptosis, and is the first drug to receive both ODD and Fast Track designation for Dercum's disease treatment.

新台北市，2024年3月3日/PRNewswire/--Caliway Biopharmaceuticals（Caliway）宣布，美国食品和药物管理局（FDA）授予CBL-514孤儿药（ODD）用于治疗德尔库姆病。CBL-514是一种脂解注射液，可通过诱导脂肪细胞凋亡来减少局部皮下脂肪，是第一种接受奇数和快速治疗德尔库姆病的药物。

'Receiving Orphan Drug Designation and Fast Track designation from the U.S. FDA is a huge milestone in CBL-514 development. Moreover, of all Dercum's disease treatment studies, CBL-514 is by far the most advanced program with the clinical efficacy being proven to reduce the size of the painful lipoma by more than 50% significantly,' said Vivian Ling, CEO of Caliway.

“从美国FDA获得孤儿药指定和快速通道指定是CBL-514开发的一个巨大里程碑。此外，Caliway首席执行官Vivian Ling说，在所有Dercum病治疗研究中，CBL-514是迄今为止最先进的项目，其临床疗效已被证明可将疼痛性脂肪瘤的大小显着减少50%以上。

'With these two designations, we look forward to accelerating CBL-514 clinical development, and becoming the first approved drug for Dercum's disease treatment with a 7-year marketing exclusivity.'

“有了这两个名称，我们期待着加速CBL-514的临床开发，并成为第一个获得批准的治疗德尔库姆病的药物，拥有7年的独家销售权。”

Caliway has completed a Phase 2 study (CBL-0201DD, NCT05387733) to evaluate CBL-514's efficacy and safety in treating Dercum's disease last year. The study results demonstrated that after treating with CBL-514, 64.5% of painful lipomas showed dimension reduction of more than 50%, 38.7% of painful lipomas showed complete clearance, with pain being reduced by 4.7 point.

Caliway去年完成了一项2期研究（CBL-0201DD，NCT05387733），以评估CBL-514治疗Dercum病的疗效和安全性。研究结果表明，用CBL-514治疗后，64.5%的疼痛脂肪瘤显示尺寸减小超过50%，38.7%的疼痛脂肪瘤显示完全清除，疼痛减轻了4.7分。

In January 2024, a placebo-controlled Phase 2b study (CBL-0202DD) to evaluate efficacy of CBL-514 for treating Dercum's disease has been approved by the U.S. FDA. The subject recruitment will be initiated in Q2 2024.

2024年1月，美国FDA批准了一项安慰剂对照的2b期研究（CBL-0202DD），以评估CBL-514治疗德尔库姆病的疗效。主题招募将于2024年第二季度开始。

About Orphan Drug Designation

关于孤儿药指定

To support the development and evaluation of new treatments for rare diseases, the U.S. FDA grants orphan drug designation to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that affects fewer than 200,000 people in the United States.

为了支持罕见病新疗法的开发和评估，美国食品和药物管理局（FDA）将孤儿药指定为药物或生物产品，以预防、诊断或治疗影响美国不到20万人的罕见病或病症。

Orphan drug designation qualifies sponsors for incentives including 7-year marketing exclusivity to sponsors of approved orphan products, federal tax credit for expenses, waiver of Prescription Drug User Fee Act (PDUFA) fees, and eligibility for regulatory assistance and guidance from the FDA in the design of a drug development plan.

孤儿药指定使赞助商有资格获得奖励，包括对批准的孤儿产品赞助商的7年独家营销，费用的联邦税收抵免，处方药用户费用法案（PDUFA）费用的豁免，以及在药物开发计划的设计中获得FDA的监管援助和指导的资格。

About Dercum's Disease

关于Dercum病

Dercum's disease is a rare disorder that is characterized by the development of painful lipomas primarily located on the trunk region, and the extremities close to the trunk. The pain is chronic (>3 months), symmetrical, often disabling, and resistant to traditional analgesics.

德尔库姆病是一种罕见的疾病，其特征是主要位于躯干区域和四肢靠近躯干的疼痛性脂肪瘤的发展。疼痛是慢性的（>3个月），对称，经常致残，并且对传统镇痛药有抵抗力。

The etiology of Dercum's disease remains unknown. There is no approved drug nor recommended standard treatment for Dercum's disease yet. The current treatment options, including surgical removal, liposuction, electrotherapy, and inflammation inhibitors, are symptomatic, and the efficacies are limited with numerous adverse events.

德尔库姆病的病因尚不清楚。目前还没有批准的药物或推荐的标准治疗德尔库姆病的方法。目前的治疗选择，包括手术切除，吸脂，电疗和炎症抑制剂，都是有症状的，并且疗效受到许多不良事件的限制。

The clinical need for Dercum's disease remains unmet.

德尔库姆病的临床需求仍未得到满足。

According to the Global Dercum's Disease Market Research Report, the global Dercum's disease treatment market size in 2021 was $11.3 billion. With a compound annual growth rate (CAGR) of 6.76%, the global market of Dercum's disease treatment in 2030 is estimated to expand to $19.95 billion.

根据全球Dercum疾病市场研究报告，2021年全球Dercum疾病治疗市场规模为113亿美元。复合年增长率（CAGR）为6.76%，预计2030年全球德尔库姆病治疗市场将扩大至195.5亿美元。

About CBL-514

关于CBL-514

CBL-514, a potentially first-in-class small-molecule drug, is an injection lipolysis drug that can induce adipocytes apoptosis and lipolysis to reduce subcutaneous adiposity in treatment areas in animal studies without causing any systematic side effects on the central nervous system, cardiovascular system, and respiratory system.

CBL-514是一种潜在的一流小分子药物，是一种注射脂解药物，可诱导脂肪细胞凋亡和脂解，以减少动物研究中治疗区域的皮下肥胖，而不会对中枢神经系统，心血管系统和呼吸系统产生任何系统性副作用。

Caliway's nonclinical studies showed that CBL-514 upregulates the apoptosis mediators caspase 3 and Bax/Bcl-2 ratio, and then induces dose-dependent adipocyte apoptosis in vivo and in vitro.

Caliway的非临床研究表明，CBL-514上调凋亡介质caspase 3和Bax/Bcl-2比率，然后在体内和体外诱导剂量依赖性脂肪细胞凋亡。

Caliway is investigating multiple indications for CBL-514, including non-invasive subcutaneous fat reduction, Dercum's disease, cellulite, and lipoma treatment.

Caliway正在研究CBL-514的多种适应症，包括非侵入性皮下脂肪减少，Dercum病，脂肪团和脂肪瘤治疗。

About Caliway Biopharmaceuticals

关于Caliway Biopharmaceuticals

Caliway Biopharmaceuticals (Caliway) is a Taiwan-headquartered, clinical-stage biopharmaceutical company driven to breakthrough drug discovery of novel small-molecule therapeutics. Listed on the emerging stock market in Taiwan (TPEX6919), Caliway aims to become an innovative pharmaceutical leader in aesthetic medicine and other diseases.

Caliway Biopharmaceuticals（Caliway）是一家总部位于台湾的临床阶段生物制药公司，致力于突破新型小分子疗法的药物发现。Caliway在台湾新兴股市（TPEX6919）上市，旨在成为美容医学和其他疾病领域的创新制药领导者。