By Fineline Cube 
China-based Zhaoke Ophthalmology Ltd (HKG: 6622), a leader in ophthalmic solutions, today announced that its proprietary melphalan formulation for the treatment of pediatric retinoblastoma has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). This designation marks a significant step toward a New Drug Investigational (IND) application in the U.S. and ensures seven years of market exclusivity upon approval.
Significance of ODD
The Orphan Drug Designation provides Zhaoke Ophthalmology with exclusive marketing rights in the U.S. for seven years post-approval. This includes both Marketing Authorization Holder (MAH) status and data exclusivity, preventing the FDA from approving other melphalan-based products for retinoblastoma, even if they are innovative. This exclusivity underscores the strong market position this designation offers.
Drug Mechanism and Indication
Melphalan, an alkylating antineoplastic agent, works by interacting with DNA to block cancer cell growth. It is currently used primarily in multiple myeloma treatment. Pediatric retinoblastoma, a rare and aggressive eye cancer affecting children under five, is diagnosed in approximately 200-300 cases annually in the U.S. and around 8,000-9,000 globally each year. Early diagnosis and treatment can lead to a global survival rate exceeding 90%.-Fineline Info & Tech