- Eli Lilly has won Food and Drug Administration approval for expanded use of Retevmo, a gene-targeted cancer drug the company acquired in its $8 billion buyout of Loxo Oncology.
- The medicine is designed for patients whose tumor growth is spurred by alterations in a gene called RET. The FDA first cleared it in May 2020, granting an accelerated approval for patients with RET fusion-positive metastatic non-small cell lung cancer.
- Now, the agency has granted full approval for that indication and broadened it to include locally advanced disease, Lilly said Wednesday. In addition, the FDA granted an accelerated approval for patients who tumors test positive for RET gene fusions, regardless of the type of cancer they have. That clearance is subject to confirmatory testing.
The FDA’s action highlights the rapid advance of targeted therapies in cancer, giving further weight to the idea of treating patients based on their cancer’s genetic makeup rather than where it’s found in body.
It’s at least the sixth time in recent years the agency has granted a “tissue-agnostic” approval to a treatment that zeroes in on a specific gene. Merck’s Keytruda led the way in 2017, followed by Bayer and Loxo’s Vitrakvi. Roche’s Rozlytrek, GlaxoSmithKline’s Jemperli and Novartis’s Taflinar and Mekinst combination have also all won approvals based on genetic mutations.
The new tissue-agnostic indication for Retevmo is for patients who have either progressed after prior treatment or don’t have a good alternative for therapy. It’s based on a subgroup of patients in a major clinical trial called Libretto that found the drug could shrink tumors, with 44% of patients experiencing a response to treatment.
Retevmo was a major draw in Lilly's decision to buy Loxo in 2019. The Indianapolis-based drugmaker highlighted it as a key growth product in its second-quarter earnings report, as sales jumped 75% to $45 million. The Loxo deal also yielded experimental drugs including pirtobrutinib, which Lilly is developing for several different indications in cancer.