Guangzhou RegeneX BioPharma's New Drug Receives FDA Orphan Drug Designation, Accelerating Global Regulatory Submissions

VCBeat (WeChat ID: vcbeat) has recently learned that Guangzhou Zaiji Pharmaceutical Technology Co., Ltd. (hereinafter referred to as “Zaiji Pharma”) announced that its clinical product, Max-40279, has officially received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). Independently developed by Zaiji Pharma’s research team, the product is indicated for the treatment of acute myeloid leukemia (AML).

Preclinical study data demonstrate that Max-40279, through potent dual inhibition of FLT3 and FGFR and high bone marrow drug concentrations, effectively overcomes resistance mediated by bone marrow FGF pathway activation associated with currently approved therapies, positioning it as a leading international clinical-stage candidate.

Dr. Arshav Lev, Vice President of Global Clinical Regulatory Affairs at the Company, stated, “This marks the first U.S. FDA regulatory milestone achieved by Guangzhou Zaiji. We will accelerate global clinical regulatory submissions for this project to deliver the most effective therapeutic agents to patients worldwide in a timely manner.”

Zaiji Pharma was established in Guangzhou in 2016 and has set up clinical operations centers in the United States and Australia. It is a pharmaceutical company dedicated to developing innovative drugs with global intellectual property rights. The company’s product development strategy centers on oral tumor immune checkpoint inhibitors targeting PD-1/PD-L1, pioneers tumor-targeted therapies, and aims ultimately at achieving all-oral combination regimens of tumor immunotherapies and targeted agents. Reportedly, Zaiji Pharma secured Series A financing from investors including Fenxiang Investment.

“Orphan drugs” refer to novel pharmaceuticals and biologics approved by the U.S. FDA for the safe and effective treatment, diagnosis, and prevention of rare diseases or disorders. Obtaining orphan drug designation in the United States accelerates FDA clinical review and approval, grants a seven-year period of exclusive marketing rights, and provides incentives such as U.S. federal tax credits. Currently, most new anti-cancer drugs in the United States are initially approved through the orphan drug pathway, with rare diseases as their indicated indications.

Furthermore, drugs approved by the U.S. FDA can relatively easily enter other markets. For instance, Article 7 of Document No. 140 issued by the China Food and Drug Administration (CFDA) states that “drugs for which applicants have simultaneously filed registration applications with drug regulatory authorities in the European Union and the United States shall be placed in a separate queue to expedite review and approval.”

Accompanying the reform of the drug review and approval system, national policy has explicitly proposed support for the research and development (R&D) of drugs and medical devices for rare diseases for the first time, which is beneficial to orphan drug R&D. According to statistics, since 2016, 14 products from seven Chinese companies, including BeiGene and CSPC Pharmaceutical Group, have obtained Orphan Drug Designation from the U.S. FDA. Zaiji Pharma is the eighth company to receive this honor.