Lilly Receives FDA Priority Review for the Selpercatinib New Drug Application

On Jan 30, 2020

Eli Lilly and Company announced that the U.S. Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) for selpercatinib (LOXO-292), for the treatment of patients with advanced RET fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. The NDA is based on data from the LIBRETTO-001 Phase 1/2 trial in RET-altered lung and thyroid cancers. The FDA has filed the NDA and set a Prescription Drug User Fee Act (PDUFA) date in the third quarter of this year.

“We are pleased the FDA granted priority review status for the NDA for selpercatinib. This represents an important step toward providing a new precision therapy for people living with certain RET-driven cancers,” said Anne White, president of Lilly Oncology. “Combined with the recent opening of our two Phase 3 selpercatinib clinical trials, we are thrilled with the positive momentum of this program and hope to deliver a practice-changing treatment to patients with RET-driven cancers as soon as possible.”

In previous regulatory actions, based on early data from the Phase 1/2 LIBRETTO-001 trial, the FDA granted selpercatinib Breakthrough Therapy Designation for treatment in people with:

Metastatic RET-fusion-positive NSCLC who require systemic therapy and have progressed following platinum-based chemotherapy and an anti-PD-1 or anti-PD-L1 therapy;
RET-mutant MTC who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment option; and
Advanced RET-fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options.

In 2019, selpercatinib received orphan drug designation for the treatment of RET fusion-positive NSCLC and for the treatment of RET fusion-positive and RET-mutant thyroid cancers including poorly differentiated thyroid cancer, undifferentiated or anaplastic thyroid cancer, MTC and locally advanced or metastatic follicular or papillary thyroid cancer.

In December of 2019, Lilly opened two selpercatinib Phase 3 trials: LIBRETTO-431 for patients with treatment-naïve RET fusion-positive NSCLC, and LIBRETTO-531 for patients with treatment-naïve RET-mutant MTC. Each trial will enroll 400 patients.