FDA Offers Accelerated Approval for 3rd Tissue-Agnostic Cancer Treatment

Regulatory NewsRegulatory News | 15 August 2019 |  By 

The US Food and Drug Administration (FDA) on Thursday granted accelerated approval to Roche’s Rozlytrek (entrectinib), a treatment for adult and adolescent patients whose cancers have the specific genetic defect neurotrophic tyrosine receptor kinase (NTRK) gene fusion and for whom there are no effective treatments.

This is the third time FDA has approved a cancer treatment based on a common biomarker across different tumor types rather than the location in the body where the tumor originated.

FDA similarly offered Bayer’s Vitrakvi accelerated approval in December 2018 as a treatment of adult and pediatric patients with solid tumors that display NTRK gene fusion, and the European Medicines Agency last month followed suit. In addition, FDA offered another tissue-agnostic accelerated approval for Merck’s Keytruda (pembrolizumab) in May 2017. FDA in 2018 also issued guidance on developing targeted therapies in low-frequency molecular subsets of a disease.

“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.

According to FDA, Rozlytrek’s ability to shrink tumors was evaluated in four clinical trials including 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.

FDA also approved the treatment on Thursday for adults with non-small cell lung cancer whose tumors are ROS1-positive and metastatic. The treatment was approved in just under six months. Clinical studies evaluated 51 adults with ROS1-positive lung cancer. 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.



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