12月23日，康宁杰瑞宣布，其自主研发的恩沃利单抗（英文通用名：Envafolimab，康宁杰瑞研发代号：KN035）获美国食品药品监督管理局 （FDA）授予孤儿药资格认定（Orphan Drug Designation，ODD），用于治疗胃癌和胃食管结合部癌（GC/GEJ）。这是继晚期胆道癌及软组织肉 瘤之后，恩沃利单抗获得的第3项孤儿药资格认定。 恩沃利单抗是皮下注射PD-L1单域抗体Fc融合蛋白。由康宁杰瑞自主研发，2016年起与思路迪医药共同开发，2020年3月30日，康宁杰瑞、思路迪 医药、先声药业三方达成战略合作，康宁杰瑞作为原研方负责生产和质量，思路迪医药负责肿瘤领域的临床开发，先声药业负责产品在中国大陆 的独家商业推广。恩沃利单抗于2021年11月在中国获批上市，适用于不可切除或转移性微卫星高度不稳定（MSI-H）或错配修复基因缺陷型 （dMMR）实体瘤。此前，已获国家药品监督管理局（NMPA）授予突破性疗法认定，用于治疗高肿瘤突变载荷（TMB-H）不可切除或转移性实 体瘤。 恩沃利单抗是皮下注射PD-L1单域抗体Fc融合蛋白。由康宁杰瑞自主研发，2016年起与思路迪医药共同开发，2020年3 ...

思路迪医药股份(01244)发布公告，旗下商业化产品恩维达(通用名：恩沃利单抗注射液，原研代号： KN035)针对胃癌和胃食管结合部癌适应症正式获得孤儿药资格认定，这是恩维达继胆管癌和软组织肉 瘤适应症后成功获批的第叁个孤儿药适应症。 此次获批基于本公司开展的恩维达晚期胃/食管胃结合部腺癌的II期临床研究展现出明确的抗肿瘤疗效， 其联合FOLFOX方案的客观缓解率达60%，疾病控制率高达100%，且安全性与耐受性良好，无导致治 疗终止或死亡的不良事件发生。 ...

Core Viewpoint - Zai Jian Pharmaceutical announced that its investigational product ZG006 has received orphan drug designation from the FDA for the treatment of neuroendocrine cancer, marking it as the first trispecific antibody targeting the DLL3 antigen with potential to be a best-in-class molecule [1] Group 1: Product Development - ZG006 is the first trispecific antibody targeting the DLL3 antigen globally [1] - The orphan drug designation will provide ZG006 with policy support for further research, registration, and commercialization in the U.S. [1] Group 2: Benefits of Orphan Drug Designation - The designation includes benefits such as tax credits for clinical trial costs, waiver of new drug application fees, and seven years of market exclusivity [1]

Core Points - The company announced that its investigational product ZG006 has received Orphan-drug Designation from the FDA for the treatment of neuroendocrine cancer [1] - ZG006 is a trispecific antibody drug developed through the company's dual/multi-specific antibody research platform [1] - The product has obtained clinical trial approval from both the FDA and China's NMPA, and has been included in breakthrough therapy designations by the respective regulatory authorities [1]

Core Insights - Wanbangde's subsidiary has made significant progress in the internationalization of its pemphigus treatment, successfully winning the first position in the 11th national drug procurement for its bromhexine hydrochloride injection [1] - The company submitted an orphan drug designation application for WP203A (alfanotide) to the FDA, which has been confirmed, marking a key step in its international strategy for pemphigus treatment [1][2] - Pemphigus is a rare autoimmune disease characterized by severe blistering and erosion of the skin and mucous membranes, primarily affecting the elderly [1][2] Industry Overview - The global prevalence of pemphigus is estimated to be between 1 to 50 cases per million, translating to approximately 70,000 to 2.1 million patients worldwide, with the most common type being pemphigus vulgaris [2] - Current treatments mainly involve corticosteroids and immunosuppressants, which have significant side effects, while biologics like rituximab and eculizumab are effective but expensive and require intravenous administration [2] - There are about 10 to 15 new drugs in clinical stages globally for pemphigus, including Wanbangde's WP203A [2] Regulatory and Market Implications - The orphan drug designation by the FDA provides various incentives for drug development, including tax credits for clinical trial costs, waiver of new drug application fees, and seven years of market exclusivity post-approval [2][3] - The global market for pemphigus drugs is expected to reach hundreds of billions of RMB, positioning the development of WP203A as a high-quality opportunity in the rare disease sector [3] - Wanbangde aims to transition from generic drugs to innovative drugs, with WP203A potentially becoming a significant player in the pemphigus treatment market due to its differentiated mechanism and international strategy [3]

Core Viewpoint - Wanbangde's subsidiary has received orphan drug designation from the FDA for WP203A (alfanorelin) to treat pemphigus [2] Group 1: Company Announcement - Wanbangde announced that its wholly-owned subsidiary, Wanbangde Pharmaceutical Group Co., Ltd., has received a recognition letter from the FDA [2] - The orphan drug designation is specifically for WP203A (alfanorelin), which is a synthetic agonist of the melanocortin-1 receptor (MC1R) [2] - Alfanorelin exerts its anti-inflammatory and immunomodulatory effects by activating the MC1R receptor [2] Group 2: Current Applications - Alfanorelin has already been successfully applied in the treatment of erythropoietic protoporphyria (EPP) [2]

Core Viewpoint - Henlius USA Inc., a wholly-owned subsidiary of the company, has received Orphan-drug Designation from the FDA for HLX43, a targeted PD-L1 antibody-drug conjugate intended for the treatment of thymic epithelial tumors (TETs) [1][2] Group 1: Product Development - HLX43 is a novel antibody-drug conjugate developed by the company, combining a DNA topoisomerase I inhibitor with a self-developed PD-L1 targeting antibody, aimed at treating advanced/metastatic solid tumors [1][2] - The Phase 1 clinical trial data for HLX43, particularly in lung cancer populations, will be updated at the World Conference on Lung Cancer (WCLC) in September 2025, showing a 37.0% objective response rate (ORR) and an 87.0% disease control rate (DCR) in patients with advanced solid tumors [2] Group 2: Regulatory and Market Implications - The Orphan-drug Designation from the FDA will facilitate the subsequent research, registration, and commercialization of HLX43 for TETs in the U.S., providing benefits such as tax credits for clinical trial costs, waiver of new drug application fees, and seven years of market exclusivity post-approval [2] - If other similar drugs for the same indication are approved before HLX43, the company must demonstrate clinical superiority to maintain the market exclusivity benefits associated with the Orphan-drug status [3]

Core Viewpoint - Henlius USA Inc., a wholly-owned subsidiary of the company, has received Orphan-drug Designation from the FDA for HLX43, a targeted PD-L1 antibody-drug conjugate for the treatment of thymic epithelial tumors (TETs) [1][2]. Group 1: Product Development - HLX43 is a novel targeted PD-L1 antibody-drug conjugate developed by the company, combining a small molecule toxin-peptide linker with a self-developed PD-L1 targeting antibody [1]. - The drug is intended for the treatment of advanced/metastatic solid tumors, particularly in patients with non-small cell lung cancer (NSCLC) who have failed prior checkpoint inhibitors and chemotherapy [2]. Group 2: Clinical Data - Phase 1 clinical trial data presented at the 2025 World Lung Cancer Conference (WCLC) showed HLX43 has a 37.0% objective response rate (ORR) and an 87.0% disease control rate (DCR) in advanced solid tumors, especially in post-line resistant NSCLC patients [2]. - Preliminary efficacy was also encouraging in patients with thymic squamous cell carcinoma (TSCC) as reported at the 2025 American Society of Clinical Oncology (ASCO) annual meeting [2]. Group 3: Regulatory and Market Implications - The Orphan-drug Designation from the FDA will facilitate the subsequent research, registration, and commercialization of HLX43 for TETs in the U.S., providing benefits such as tax credits for clinical trial costs, exemption from new drug application fees, and seven years of market exclusivity post-approval [2]. - If another drug for the same indication is approved before HLX43, the company must demonstrate clinical superiority to maintain the market exclusivity benefits associated with the Orphan-drug status [3].

Core Viewpoint - Henlius USA Inc., a wholly-owned subsidiary of the company, has received Orphan-drug Designation from the FDA for HLX43, a targeted PD-L1 antibody conjugate for the treatment of thymic epithelial tumors (TETs) [1] Group 1 - The FDA has granted Orphan-drug Designation for HLX43, indicating its potential significance in treating a rare condition [1] - HLX43 is specifically developed as a targeted therapy for thymic epithelial tumors, which are rare types of tumors [1]

Core Viewpoint - Vanda Pharmaceuticals Inc. has received Orphan Drug Designation from the FDA for VGT-1849B, a selective JAK2 inhibitor aimed at treating polycythemia vera (PV) [1][5]. Group 1: Product Overview - VGT-1849B is a selective peptide nucleic acid-based JAK2 inhibitor designed to target JAK2 mRNA, thereby reducing JAK2 protein production and downstream signaling associated with PV [3][8]. - The prevalence of PV in the U.S. is estimated to affect 44 to 57 per 100,000 people, with over 95% of patients harboring the JAK2 V617F mutation [2][9]. - VGT-1849B utilizes a novel backbone chemistry, OliPass Peptide Nucleic Acid (OPNA), which enhances cell permeability and RNA affinity [2][8]. Group 2: Mechanism of Action - By selectively targeting JAK2 mRNA, VGT-1849B effectively reduces JAK2-driven cell proliferation and suppresses hematopoiesis, leading to decreased production of red blood cells, neutrophils, platelets, and lymphocytes [3][4]. - The drug aims to provide a favorable safety profile by avoiding off-target effects commonly associated with other JAK inhibitors [4]. Group 3: Market Context - Current JAK2 inhibitors on the market, such as Jakafi®, Inrebic®, Ojjaara®, and Vonjo®, are not solely selective to JAK2, which can lead to increased toxicity [4]. - If approved, VGT-1849B could offer targeted efficacy with an improved safety profile and convenient infrequent dosing, addressing a significant unmet medical need in the treatment of PV [5]. Group 4: Company Background - Vanda Pharmaceuticals Inc. is focused on developing innovative therapies to meet high unmet medical needs and improve patient lives [7].