FDA Grants Accelerated Approval to Larotrectinib for NTRK+ Cancers

The FDA has granted an accelerated approval to larotrectinib for the treatment of adult and pediatric patients with solid tumors that have an NTRK

gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment.

The approval is based on findings from patients with TRK-positive tumors enrolled across 3 clinical trials. In results published in the New England Journal of Medicine (NEJM)

in February 2018, larotrectinib induced an objective response rate of 75% (95% CI, 61-85) by independent review and 80% (95% CI, 67-90) by investigator assessment in 55 evaluable patients. Per the independent assessment, there were 7 (13%) complete responses, 34 (62%) partial responses, and 7 (13%) patients with stable disease.

At 1 year, 71% of responses were ongoing. More than half (55%) of patients remained progression-free at 1 year. The median duration of response had not been reached after a median follow-up of 8.3 months. The same was true for median progression-free survival after a median follow-up of 9.9 months.

The FDA reviewed data from 55 adult and pediatric patients with TRK

fusion—positive cancers enrolled across a phase I adult trial, the phase II NAVIGATE trial, and the phase I/II SCOUT pediatric trial. The data cutoff for the NEJM

findings was July 17, 2017.

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