Sequential orphan drug designations for the same candidate
from pancreatic and gastric cancer to small cell lung cancer
Onconic Therapeutics announced on the 24th that its next-generation dual-target synthetic lethal anticancer drug candidate, Nesuparib, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) as a treatment for small cell lung cancer (SCLC). Nesuparib had previously received orphan drug designation for pancreatic cancer in 2021 and for gastric cancer in 2025, and has now added small cell lung cancer to the list.
Small cell lung cancer is a hard-to-treat malignancy characterized by rapid proliferation, early metastasis, and a high recurrence rate, and it is an area where treatment options are limited once the disease relapses after first-line therapy. Nesuparib is a dual-mechanism synthetic lethal anticancer agent that simultaneously inhibits PARP, an enzyme protein, and tankyrase (TNKS). It is designed to concurrently target inhibition of DNA damage repair and blockade of tumor growth and adaptive signaling.
Small cell lung cancer is a tumor type that exhibits high genomic instability and dependence on replication stress due to loss of the TP53 and RB1 genes. Therapeutic strategies targeting the DNA damage response are therefore considered to have strong rationale. New approaches such as combination regimens with immune checkpoint inhibitors and DLL3-targeted therapies are currently being explored, but treatment options that can induce a durable response after relapse remain limited.
Onconic Therapeutics is conducting a phase 2 clinical trial of Nesuparib in pancreatic cancer, a phase 2 clinical trial in ovarian cancer in combination with Vegzelma, an investigator-initiated phase 2 clinical trial in endometrial cancer in combination with Keytruda, and a phase 1b/2 clinical trial in gastric cancer. A company representative said, "We plan to continue expanding the indications of Nesuparib, focusing on tumor types with high dependence on replication stress and DNA damage response."
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