publication date: Nov. 30, 2018

FDA approves second drug for a site-agnostic indication; larotrectinib was tested across 17 cancer types

By Paul Goldberg

Vitrakvi (larotrectinib) aims to treat a very small group of people—some say fewer than 3,000 new patients a year in the U.S. And since these patients have diseases that span multiple tumor sites, finding them isn’t easy.

Yet, this drug’s significance isn’t based on the size of the population it treats.

As the second agent to receive an accelerated approval for a site-agnostic indication, the tyrosine kinase inhibitor co-developed by Loxo Oncology  Inc. and Bayer and approved by FDA on Nov. 26, strengthens the rationale for reliance on next-generation sequencing as a basis for treatment decisions and sends a loud signal that FDA is equipped to usher site-agnostic targeted therapies onto the U.S. market.

“It is true that the number of patients in the absolute sense is estimated to be relatively small—in the several thousand range per year for the U.S.—but the benefit to those patients is dramatic and highly meaningful,” said David Hyman, chief of the Early Drug Development Service at Memorial Sloan Kettering Cancer Center and a global principal investigator for a larotrectinib clinical trial. “So, I think the importance for these patients stands on its own, including for patients with cancer types who are historically under-studied and under-resourced in the development pipeline: pediatric cancers, sarcomas, other rare cancers.”

In clinical trials of patients with TRK fusion cancer, larotrectinib demonstrated an ORR of 75 percent (N=55) (95% CI, 61%, 85%), including a 22 percent complete response rate.

“Another important aspect of this approval is that it provides a second tumor-agnostic indication, which, on top of MSI, further improves the overall utility … Continue reading FDA approves second drug for a site-agnostic indication; larotrectinib was tested across 17 cancer types

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