FDA on Thursday approved Roche's cancer drug designed to target a tumor's genetic driver rather than the tissue where the tumor originated.
FDA in a release said Roche's Rozlytrek is the third cancer drug to gain approval "based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated." Merck's Keytruda in 2017 became the first cancer treatment FDA approved to target a tumor's biomarker instead of the type of tissue from which the tumor originated. The approval marked a major step in treating cancer with precision medicine.
FDA approves cancer drug to target cancer tumors with rare genetic mutations
FDA granted Roche an accelerated approval for Rozlytrek to treat adolescents and adults who have solid cancer tumors with rare genetic mutations, called neurotrophic tyrosine receptor kinase (NTRK) gene fusions and who do not respond to other treatments. It is difficult to conduct tests for individual NTRK gene fusion mutations. Researchers have identified NTRK gene fusion mutations in several solid tumor types, including breast, colorectal, non-small cell lung cancers, and pancreatic.
FDA said Rozlytrek's ability to shrink tumors was assessed based on data from four clinical trials involving 54 adults with NTRK fusion-positive tumors in locations such as the breast, colon/rectum, lung, salivary gland, and thyroid. FDA said the data showed 57% of patients, or 31 patients, experienced substantial tumor shrinkage, including 7.4% of patients who experienced a complete disappearance of their tumor. Among the patients with tumor shrinkage, 61% experienced tumor shrinkage for at least nine months, FDA said.
The agency determined the Rozlytrek's effectiveness for adolescents based on the adult data from the four clinical trials, according to Richard Pazdur, director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. "[S]afety was demonstrated in 30 pediatric patients," Pazdur said.
Some of Rozlytrek's common side effects include diarrhea, constipation, and fatigue, according to FDA. The agency said the "most serious" side effects of the drug include congestive heart failure, central nervous system effects, and damage to the liver.
FDA also approved Rozlytrek to treat adults with non-small cell lung cancer whose tumors have a genetic mutation, called ROS1 gene fusion, and have spread to other parts of the body. FDA said clinical trials involving 51 patients with ROS1-positive lung cancer showed 78% of the patients, or 40 patients, experienced tumor shrinkage, including 5.9% of patients who experienced complete tumor disappearance. Among the patients with tumor shrinkage, 55% experienced tumor shrinkage for at least 12 months, FDA said.
FDA Acting Commissioner Ned Sharpless said, "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."
Sandra Horning, chief medical officer at Roche's Genentech division, in a statement, said, "Rozlytrek's FDA approval for two rare types of cancer is an important advance for patients, combining a targeted medicine and genomic testing to bring this new treatment option to patients who are waiting. Rozlytrek is the first FDA-approved treatment that selectively targets both ROS1 and NTRK fusions and, importantly, has also shown responses in these rare cancer types that have spread to the brain."
Roche did not provide details on the Rozlytrek's price and did not immediately respond to a request for comment, Reuters reports (Maddipatla/Jagadeesh Babu, Reuters, 8/15; Dearment, MedCity News, 8/16; Paavola, Becker's Hospital Review, 8/16; FDA release, 8/15).