US-based Eli Lilly and Company (Lilly) has secured the US Food and Drug Administration (FDA) approval for Retevmo (selpercatinib), to treat a type of non-small cell lung cancer (NSCLC) and medullary thyroid cancer (MTC).

The US FDA indicated Retevmo for the treatment for metastatic rearranged during transfection (RET) NSCLC in adults, metastatic RET-mutant MTC in adults and paediatric patients aged 12 years and above who require systemic therapy, and metastatic RET fusion-positive thyroid cancer.

Retevmo is a selective RET kinase inhibitor that has the potential to affect both tumour cells and healthy cells, and can also result in side effects. The drug is prescribed as an oral medicine in 120 mg or 160 mg formulation based on weight and is advised to take twice daily.

The regulatory approval for Retevmo is based on LIBRETTO-001 clinical trial

Lilly has secured approval for Retevmo under FDA accelerated approval regulations, based on objective response rate (ORR) and duration of response (DoR) data from the LIBRETTO-001 Phase 1/2 trial.

LIBRETTO-001 trial lead investigator Alexander Drilon said: “In the clinical trial, we observed that the majority of metastatic lung cancer patients experienced clinically meaningful responses when treated with selpercatinib, including responses in difficult-to-treat brain metastases.

“The approval of selpercatinib marks an important milestone in the treatment of NSCLC, making RET-driven cancers now specifically targetable in the same manner as cancers with activating EGFR and ALK alterations, across all lines of therapy. I am pleased that patients with these RET-driven cancers have this newly approved option.”

The single-arm, multi-centre Phase 1/2 LIBRETTO-001 trial enrolled both treatment-naive patients and heavily pretreated patients with a type of advanced solid tumours including RET fusion-positive NSCLC, RET-mutant MTC, RET fusion-positive thyroid cancer.

The major efficacy outcomes of the study include ORR and DoR, as evaluated by a blinded independent review committee, and the pre-specified secondary endpoints include central nervous system (CNS) ORR and CNS DoR.