Core Viewpoint - The company, Zhaoke Ophthalmology-B (06622.HK), has received Orphan Drug Designation (ODD) from the FDA for its proprietary formulation, Mephalan, aimed at treating retinoblastoma (RB), a rare childhood eye cancer [1] Group 1: Regulatory Approval - The ODD provides a clear regulatory pathway for the company to submit a new drug clinical trial application in the U.S. [1] - If Mephalan is successfully developed and approved, the company will enjoy seven years of exclusive market rights in the U.S. post-approval [1] - The exclusivity includes marketing authorization holder (MAH) status and data exclusivity, preventing the FDA from approving any other RB indication products based on Mephalan, even if the formulation is modified [1] Group 2: Drug Mechanism and Current Use - Mephalan is an alkylating agent that interferes with DNA replication and cell division, blocking the growth and spread of cancer cells [1] - Currently, Mephalan is primarily used as a pre-treatment for autologous hematopoietic stem cell transplantation in multiple myeloma patients or for palliative treatment in cases where oral therapy is not applicable [1]
兆科眼科-B(06622.HK)：用于治疗儿童视网膜母细胞瘤的美法仑取得美国FDA孤儿药资格认证