Onconic Therapeutics announced on the 24th that its novel anti-cancer candidate, 'Nesuparib', has been designated as an Orphan Drug (ODD) for the treatment of small cell lung cancer (SCLC) by the U.S. Food and Drug Administration (FDA).
This marks the third ODD designation for Nesuparib, following its previous recognition for pancreatic cancer in 2021 and stomach cancer in 2025. With this latest development, Onconic Therapeutics has reaffirmed the potential of Nesuparib as a versatile, multi-cancer therapeutic.
A Critical Lifeline for Intractable Cancer
The FDA’s Orphan Drug Designation is granted to treatments for rare diseases that demonstrate significant medical potential and a high necessity for development. Upon receiving final regulatory approval, the ODD status provides the drug with seven years of market exclusivity, along with various tax incentives and clinical trial support.
Small cell lung cancer is a notoriously aggressive form of the disease, characterized by rapid tumor proliferation, early metastasis, and a high rate of recurrence. Because treatment options are severely limited once the cancer returns following first-line therapy, improving long-term survival rates remains one of the most pressing challenges in oncology.
Dual-Action Mechanism: Inhibiting PARP and Tankyrase
Nesuparib distinguishes itself through a unique dual-action mechanism. It simultaneously inhibits 'PARP' (Poly ADP-ribose polymerase), an enzyme critical for repairing single-strand DNA damage, and 'Tankyrase' (TNKS), which regulates several vital signaling pathways within the body, including the Wnt and Hippo pathways.
"Small cell lung cancer is known to exhibit increased activity in signaling pathways like Wnt and Hippo, while also relying heavily on DNA damage responses during treatment," stated a representative from Onconic Therapeutics. "The FDA’s positive evaluation suggests that Nesuparib can effectively block these pathways, potentially offering a new alternative for patients struggling with recurrent and treatment-resistant cases."
