Onconic Therapeutics (hereinafter referred to as "Onconic"), a subsidiary of Jeil Pharmaceutical specializing in new drug development, announced that its next-generation anticancer drug candidate Nesuparib has been designated as an Orphan Drug (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of gastric and gastroesophageal junction cancer.
Onconic stated on the 18th, “With this orphan drug designation, Nesuparib has once again been recognized for its technological innovation in the field of gastric cancer treatment.” Nesuparib had previously received orphan drug designation from both the FDA and South Korea’s Ministry of Food and Drug Safety (MFDS) in 2021 for pancreatic cancer.
Nesuparib is a next-generation targeted anticancer therapy that employs a dual inhibition mechanism targeting both Poly ADP-ribose polymerase (PARP) and Tankyrase. PARP is an enzyme involved in DNA damage repair, aiding the survival of cancer cells. Tankyrase plays a crucial role in the Wnt/β-catenin signaling pathway, which regulates cancer cell growth and metastasis. By inhibiting Tankyrase, the proliferation of cancer cells can be effectively suppressed. Onconic expects that this dual-targeting approach will overcome the limitations of existing PARP inhibitors and provide efficacy even in hard-to-treat cancers.
Industry experts highlight the significance of Nesuparib's FDA orphan drug designation, noting that the approval rate for orphan drug designation is only 17.6%. Given that a high percentage of FDA-approved new drugs were previously designated as orphan drugs, Nesuparib's chances for final market approval and commercialization are gaining attention.
FDA orphan drug designation provides several advantages for drug development and commercialization, including fast-track review, accelerated approval, exemption from new drug application (NDA) review fees, R&D funding support, and extended market exclusivity. These benefits can expedite the new drug’s development and market entry.
A representative from Onconic stated, “The fact that the same drug candidate has received orphan drug designation for two different cancer types demonstrates Nesuparib's high technological potential and anticancer efficacy.” They further emphasized, “We will continue our research and development efforts to provide new treatment options for gastric cancer patients.”
