Zhaoke Ophthalmology Limited (“Zhaoke Ophthalmology”, “Zhaoke” or the “Company”, SEHK: 6622), a leading ophthalmic pharmaceutical company, is pleased to announce that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation (“ODD”) to its proprietary formulation of melphalan for the treatment of pediatric retinoblastoma (“RB”), a rare pediatric eye cancer. 

The FDA's decision recognizes Melphalan’s potential in addressing a critical unmet medical need. Melphalan, an alkylating chemotherapeutic agent, exerts its anti-cancer effects by chemically modifying DNA strands within tumor cells. This process creates cross-links that disrupt DNA replication and transcription, selectively targeting rapidly dividing cancer cells while offering potential advantages for localized administration in RB.

Securing ODD delivers significant strategic advantages for Zhaoke Ophthalmology. It establishes a clear regulatory pathway toward Investigational New Drug (“IND”) submission in the U.S. More substantially, if Melphalan is successfully developed and approved, the Company would become eligible for seven years of U.S. market exclusivity upon New Drug Application (“NDA”) approval. This comprehensive protection encompasses marketing authorization holder (“MAH”) status, data exclusivity, and crucially, prevents FDA approval of any other melphalan-based product for the RB indication during this period – regardless of formulation innovations. While no competing therapies are currently approved for RB indication, Zhaoke remains committed to rapid development to maintain its potential first-mover advantage.

Dr. Li Xiaoyi (Benjamin), Chairman of the Board, Executive Director and CEO of Zhaoke Ophthalmology, commented, “We are thrilled to have received the FDA’s ODD for our innovative pediatric retinoblastoma treatment. This achievement marks a significant milestone in our plans to enter the U.S. market. The Orphan Drug Act grants us seven years of market exclusivity, providing a powerful advantage and encouraging us to invest further in the development and commercialization of this groundbreaking therapy. Critically, this designation brings us closer to our mission of supporting children and families affected by this rare and devastating eye cancer.”

About Melphalan

Melphalan is an alkylating agent that exerts its anticancer activity by reacting with DNA and interfering with DNA replication and cell division. This stops the growth and spread of cancer cells in the body. Currently, Melphalan is given as the conditioning regimen prior to autologous stem cell transplantation in patients with multiple myeloma, or as the palliative treatment of multiple myeloma if oral therapy is not appropriate.

About Pediatric Retinoblastoma

Pediatric retinoblastoma is a rare eye cancer that primarily affects children under the age of five. In the United States, approximately 200 to 300 cases are diagnosed annually (American Cancer Society, 2023). Globally, the incidence is approximately one in 15,000 to 18,000 live births, resulting in roughly 8,000 to 9,000 new cases each year (World Health Organization, 2024). The overall survival rate exceeds 90% worldwide when diagnosis is made promptly and appropriate care is provided.

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