FDA recently approved the third oncology drug in the United States that targets a key genetic driver of cancer, rather than a specific tumor.
The agency granted accelerated approval to entrectinib (Rozlytrek. Genentech), a treatment for adult and adolescent patients whose cancers have the specific genetic defect, NTRK (neurotrophic tyrosine receptor kinase) gene fusion and for whom there are no effective treatments.
Rozlytrek was also approved to treat adults with non-small cell lung cancer whose tumors are ROS1-positive (mutation of the ROS1 gene) and has spread to other parts of the body.
The approval marks a new paradigm in the development of cancer drugs that are “tissue agnostic,” FDA said in a statement.
The previous tissue agnostic indications approved by the FDA were pembrolizumab (Keytruda, Merck) for tumors with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in 2017 and larotrectinib (Vitrakvi, Loxo Oncology) for NTRK gene fusion tumors in 2018.
“We are in an exciting era of innovation in cancer treatment as we continue to see development in tissue agnostic therapies, which have the potential to transform cancer treatment. We’re seeing continued advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine,” said FDA Acting Commissioner Ned Sharpless, MD, in a statement from the agency.
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“Using the FDA’s expedited review pathways, including breakthrough therapy designation and accelerated approval process, we’re supporting this innovation in precision oncology drug development and the evolution of more targeted and effective treatments for cancer patients,” Sharpless added.
The ability of Rozlytrek to shrink tumors was evaluated in 4 clinical trials, studying 54 adults with NTRK fusion-positive tumors. The proportion of patients with substantial tumor shrinkage (overall response rate) was 57%, with 7.4% of patients having complete disappearance of the tumor.
Among the 31 patients with tumor shrinkage, 61% had tumor shrinkage persist for nine months or longer. The most common cancer locations were the lung, salivary gland, breast, thyroid and colon/rectum.
For the non-small cell lung cancer indication, 51 adults with ROS1-positive lung cancer were studied. The overall response rate was 78%, with 5.9% of patients having complete disappearance of their cancer. Among the 40 patients with tumor shrinkage, 55% had tumor shrinkage persist for 12 months or longer, according to FDA.