Core Viewpoint - The company has received Orphan Drug Designation (ODD) from the FDA for its proprietary formulation, Mephalan, aimed at treating retinoblastoma (RB), a rare pediatric eye cancer [1] Group 1 - The ODD provides a clear regulatory pathway for the company to submit a New Drug Application (NDA) in the United States [1] - If Mephalan is successfully developed and approved, the company will enjoy seven years of exclusive market rights in the U.S. following the NDA approval [1] - The exclusivity includes marketing authorization holder (MAH) status and data exclusivity, ensuring that the FDA cannot approve any other RB indication products based on Mephalan, even if the formulation is modified [1]

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]

Zhaoke Ophthalmology Limited （於英屬處女群島註冊成立並於開曼群島存續的有限公司） （股份代號：6622） 兆科眼科有限公司 自 願 性 公 告－ 用於治療兒童視網膜母細胞瘤的美法侖 取得美國FDA孤兒藥資格認證 本 公 告 乃 由 兆 科 眼 科 有 限 公 司（「本公司」）董 事 會（「董事會」）自 願 作 出。 香港交易及結算所有限公司及香港聯合交易所有限公司對本公告的內容概不 負 責，對 其 準 確 性 或 完 整 性 亦 不 發 表 任 何 聲 明，並 明 確 表 示，概 不 對 因 本 公 告 全部或任何部份內容而產生或因倚賴該等內容而引致的任何損失承擔任何責 任。 取 得ODD為 在 美 國 提 交 新 藥 臨 床 試 驗 申 請（「新藥試驗申請」）建 立 清 晰 的 監 管 路 徑。再 者，如 果 美 法 侖 獲 成 功 研 發 並 獲 批 准，本 公 司 將 在 新 藥 上 市 申 請（「新 藥 申 請」）批 准 後 享 有 七 年 美 國 市 場 獨 家 權 利。此 項 完 善 保 護 涵 蓋 上 市 許 可 持 有 人 （marketing authoriza ...