NEW YORK – Cambridge, Massachusetts-based Blueprint Medicines said on Wednesday that it has submitted a new drug application to the US Food and Drug Administration for pralsetinib for the treatment of patients with advanced, RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer.
Blueprint is also seeking FDA approval for pralsetinib in RET-positive non-small cell lung cancer and filed an NDA for that indication in early April.
Both NDAs are supported by results from the Phase I/II ARROW trial, which is evaluating pralsetinib in patients with a variety of RET fusion-positive solid tumors. While the study is still ongoing, data presented at the American Society of Clinical Oncology's virtual annual meeting last month was encouraging: among 11 response-evaluable RET fusion-positive thyroid cancer patients, the overall response rate was 91 percent, and the drug was shown to be tolerable. Previously reported responses among patients with RET fusion-positive medullary thyroid cancer were similarly encouraging.
Pralsetinib is an investigational oral agent developed to selectively target RET alterations. In preclinical studies, the drug demonstrated the potential to suppress primary and secondary tumor mutations, overcoming clinical resistance to RET-inhibition. The FDA has granted breakthrough therapy designation for the drug in NSCLC and is evaluating the thyroid cancer NDA under its Real-Time Oncology Review pilot program, which aims to increase efficiency in the review process.
"Pralsetinib has broad potential to address the medical needs of patients with RET-altered cancers, who have not traditionally benefited from targeted therapy even though their tumors have a known disease driver," Andy Boral, Blueprint's CMO, said in a statement. "We are working closely with the FDA and aim to bring this promising treatment to patients as expeditiously as possible."
If approved, pralsetinib would compete with Lilly's RET-targeting agent selpercatinib (Retevmo). The FDA granted accelerated approval to selpercatinib in May for patients with RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer, and RET fusion-positive thyroid cancer.