The approval includes an indication for pediatric patients over 12 years of age with NTRK-fusion-positive tumors based on information obtained primarily in adults.

The U.S. Food and Drug Administration has recently granted approval to an oncology drug named Rozlytrek (entrectinib) that focuses on treating adult and adolescent patients with cancers that have neurotrophic tyrosine receptor kinase (NTRK) gene fusion.

Reportedly, this is the third time the FDA has approved a cancer treatment depending on a common biomarker across various types of tumors rather than for a specific body part where the tumor originates. The approval also marks a new paradigm in the development of cancer drugs that are tissue agnostic.

Speaking on the approval, Ned Sharpless, M.D., FDA Acting Commissioner, said that the world is currently in an era of developing innovative cancer treatments and their agency is continuously witnessing growth in tissue agnostic therapies that have the potential to transform cancer treatments.

He claims that their agency is supporting this innovation in precision oncology drug development by means of expedited review pathways comprising of advanced therapy designation and improved approval procedures. The FDA is also supporting the evolution of more effective and targeted treatments for cancer patients, claims Sharpless.

Commenting further, he said that their agency will remain committed towards supporting the advancement of targeted innovations in oncology therapeutics and across disease types based on its growing know-how of underlying biology of diseases.

For the uninitiated, the FDA’s prior approved tissue agnostic indications included pembrolizumab for tumors with MSI-H (microsatellite instability-high) or dMMR (mismatch repair deficient) tumors in 2017 and larotrectinib for tumors with NTRK gene fusion in 2018.

Meanwhile, the recent approval includes an indication for pediatric patients over 12 years of age who have NTRK-fusion-positive tumors based on efficacy information obtained primarily in adults. Additionally, Rozlytrek was approved for the treatment of adults with non-small cell lung cancer whose tumors are ROS1-positive and has spread to other parts of the body.

https://www.fda.gov/news-events/press-announcements/fda-approves-third-oncology-drug-targets-key-genetic-driver-cancer-rather-specific-type-tumor