Core Viewpoint - Suzhou Zelgen Biopharmaceutical Co., Ltd. announced that its injectable ZG006 has received Orphan-drug Designation from the FDA for the treatment of neuroendocrine cancer, which may provide certain policy support for its subsequent development and commercialization in the U.S. [2][5] Group 1: Drug Information - ZG006 (INN name: alveltamig) is a trispecific antibody drug developed through the company's dual/multi-specific antibody platform, and it has received clinical trial approval from both the FDA and China's NMPA [3]. - ZG006 is the first-in-class molecule targeting DLL3, with potential to become a best-in-class drug, as it connects tumor cells and T cells to enhance tumor cell killing [3]. Group 2: Regulatory and Development Implications - The Orphan-drug Designation allows ZG006 to benefit from various policy supports, including tax credits for clinical trial costs, exemption from new drug application fees, and seven years of market exclusivity [5]. - The company must still engage with the FDA regarding subsequent clinical trials and registration plans, with the success of these efforts remaining uncertain [2][6]. Group 3: Clinical Research Updates - The company presented clinical research data for ZG006 and ZG005 at the 2025 European Society for Medical Oncology (ESMO) annual meeting, indicating ongoing progress in its clinical development [4].

Core Viewpoint - ZaiJing Pharmaceutical (688266) has received orphan drug designation from the FDA for its investigational product ZG006, intended for the treatment of neuroendocrine cancer, which will facilitate its subsequent research, registration, and commercialization in the U.S. [1] Group 1 - The orphan drug designation provides ZaiJing Pharmaceutical with various policy supports, including tax credits for clinical trial costs [1] - The company will be exempt from new drug application fees as a result of this designation [1] - ZG006 will enjoy seven years of market exclusivity, independent of patent considerations [1]

Core Viewpoint - The company’s subsidiary has received orphan drug designation from the FDA for WP203A (afamelanotide) to treat pemphigus, which is a rare autoimmune blistering disease [1][2]. Summary by Sections Qualification Recognition - The company submitted an application for orphan drug designation for WP203A to the FDA in August 2025, under application number DRU-2024-10630, and received confirmation of approval based on the Federal Food, Drug, and Cosmetic Act [1]. Drug Overview - Pemphigus is characterized by widespread blistering and erosion of the skin and mucous membranes, primarily affecting the elderly due to immune system dysfunction. Symptoms include severe itching, redness, and painful lesions, with the potential for life-threatening complications [1]. Impact of Orphan Drug Designation - The orphan drug designation for WP203A is expected to accelerate the company’s international drug development efforts. This designation provides various incentives, including tax credits for clinical trial costs, waiver of new drug application fees, and seven years of market exclusivity post-approval [2].

Core Viewpoint - Wanbangde's subsidiary has received orphan drug designation from the FDA for WP203A (afamelanotide) to treat pemphigus, a rare autoimmune blistering disease, which may accelerate the company's international drug development efforts [1][3]. Group 1: FDA Orphan Drug Designation - The FDA granted orphan drug status to WP203A for the treatment of pemphigus, providing various incentives for drug development, including tax credits for clinical trial costs and a seven-year market exclusivity post-approval [1][2]. - Wanbangde submitted the orphan drug designation application for WP203A on August 2025, which was confirmed by the FDA [2]. Group 2: Mechanism and Current Treatment Landscape - Afamelanotide acts as a synthetic agonist of the MC1R receptor, demonstrating anti-inflammatory and immunomodulatory effects, and has been successfully used in treating erythropoietic protoporphyria (EPP) [2]. - Current treatments for pemphigus primarily include systemic corticosteroids and monoclonal antibodies, which have high relapse rates and severe side effects, indicating an unmet clinical need [2]. Group 3: Future Development and Uncertainties - Following the orphan drug designation, Wanbangde must conduct clinical trials for WP203A in accordance with FDA regulations, with uncertainties surrounding trial approval, results, and subsequent market application [3].

Core Insights - Corning Jereh Pharmaceutical-B (09966) announced that JSKN003 has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA) for the treatment of platinum-resistant recurrent epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer (collectively referred to as PROC) regardless of HER2 expression levels [1] Group 1 - JSKN003 has been approved by the FDA to conduct a Phase II clinical trial for treating PROC with no restrictions on HER2 expression levels [1] - The drug has also received Breakthrough Therapy Designation from the National Medical Products Administration (NMPA) for both PROC and colorectal cancer (CRC) [1] - Additionally, JSKN003 has been granted Orphan Drug Designation by the FDA for gastric/gastroesophageal junction cancer (GC/GEJ) [1] Group 2 - The Phase III clinical trial for JSKN003 in treating PROC is currently progressing smoothly in China [1] - The FTD designation further underscores the confidence of international regulatory bodies in the clinical potential of JSKN003 and its significance as a novel therapeutic candidate [1]

Core Insights - Suzhou Ribobio Technology Co., Ltd. and its subsidiary Ribocure Pharmaceuticals AB announced that the European Medicines Agency (EMA) granted orphan drug designation for their siRNA candidate drug RBD1016, aimed at treating hepatitis D virus (HDV) infection [1][2] Group 1: Orphan Drug Designation - The orphan drug designation by EMA is intended to encourage therapies for rare diseases with a prevalence of less than 5 in 10,000, which pose a serious threat to life or cause chronic debilitating conditions [1] - This designation provides innovative drugs with better regulatory pathways and commercialization incentives, allowing for faster patient access [1] Group 2: Clinical Development - RBD1016 is being developed using Ribobio's proprietary RiboGalSTARTM liver-targeted delivery platform, which has demonstrated safety, efficacy, and long-lasting effects through multiple clinical studies [1] - The drug is currently advancing through Phase II clinical trials for both hepatitis B and D globally [1] Group 3: Company Statements - The co-CEO and Global R&D President of Ribobio stated that the orphan drug designation is a significant milestone for RBD1016, enhancing its development and commercialization prospects [2] - The company is committed to advancing the clinical research of RBD1016 to provide new treatment options for patients suffering from this rare disease [2]

Core Viewpoint - Fosun Pharma's subsidiary, Shanghai Fuhong Hanlin Biotechnology Co., Ltd., has received Orphan-drug Designation from the FDA for its investigational drug HLX43, aimed at treating thymic epithelial tumors (TETs) [1] Group 1: FDA Orphan-drug Designation - The Orphan-drug Designation for HLX43 will facilitate its research, registration, and commercialization in the U.S. by providing various policy supports [1] - Benefits include tax credits for clinical trial costs, exemption from new drug application fees, and a seven-year market exclusivity without patent influence [1] Group 2: Market Considerations - If other drugs with the same indication are approved before HLX43, the company must demonstrate HLX43's superior efficacy in clinical settings to maintain the market exclusivity benefits [1]

Core Viewpoint - Fosun Pharma's subsidiary, Shanghai Fuhong Hanlin Biotechnology Co., Ltd., has received Orphan-drug Designation from the FDA for its investigational drug HLX43, aimed at treating thymic epithelial tumors (TETs) [1] Group 1: FDA Orphan-drug Designation - The Orphan-drug Designation for HLX43 will facilitate its subsequent research, registration, and commercialization in the U.S. by providing certain policy supports [1] - Benefits of the designation include tax credits for clinical trial costs, exemption from new drug application fees, and seven years of market exclusivity unaffected by patent status [1] Group 2: Market Considerations - If other drugs with the same indication are approved before HLX43, the company must demonstrate HLX43's superior efficacy in clinical settings to maintain the market exclusivity benefits associated with the orphan drug status [1]

Core Viewpoint - Fosun Pharma's subsidiary, Shanghai Fuhong Hanlin Biotechnology Co., Ltd., has received Orphan-drug Designation from the FDA for its investigational drug HLX43, aimed at treating thymic epithelial tumors (TETs) [1] Group 1: Drug Development - HLX43 is a targeted PD-L1 antibody-drug conjugate (ADC) developed by Fuhong Hanlin, combining a novel DNA topoisomerase I inhibitor with a self-developed PD-L1 targeting antibody [1] - The drug is intended for the treatment of advanced/metastatic solid tumors [1]

Core Viewpoint - Kangfang Biotech (09926) has received Orphan Drug Designation from the FDA for its novel anti-CD47 humanized IgG4 monoclonal antibody, Lefacitinib (AK117), for the treatment of acute myeloid leukemia (AML) [1][2] Group 1: Company Developments - Kangfang Biotech's stock rose nearly 3% in early trading, currently up 1.98% at HKD 133.5, with a trading volume of HKD 150 million [1] - The Orphan Drug Designation will provide various incentives for the development and commercialization of Lefacitinib in the U.S., including tax credits for clinical trial costs, waiver of BLA application fees, and potential R&D funding [1] Group 2: Product Details - Lefacitinib specifically binds to CD47 expressed on tumor cells, blocking its interaction with the SIRPα receptor, which enhances macrophage phagocytosis of tumor cells and inhibits tumor growth [2] - The drug's unique design prevents red blood cell aggregation, significantly improving its safety and efficacy compared to other drugs targeting the same pathway, enhancing therapeutic convenience [2]