FDA Approves Genentech's Rozlytrek for Two Rare Types of Cancer

FDA Approval

Genentech’s personalized cancer medication Rozlytrek (entrectinib) won approvals for two rare types of cancer from the U.S. Food and Drug Administration (FDA).

The FDA approved Rozlytrek for the treatment of adults with ROS1-positive, metastatic non-small cell lung cancer (NSCLC). Also, Rozlytrek secured accelerated approval for the treatment of solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy.

Click to view our 2019 hotbed maps of biotech, pharma and medical device company clusters in the USA

This is the first treatment approved for a therapy designed to target both ROS1 and NTRK and also shows a response in cancer that has spread to the brain, Genentech, a subsidiary of Swiss pharma giant Roche, said late Thursday. Rozlytrek represents a unique, tumor-agnostic approach to cancer treatment, which means that it can target a specific cancer-causing genetic rearrangement, such as NTRK, regardless of a tumor’s site of origin, the company said. NTRK gene fusions have been identified in a range of hard-to-treat solid tumor types, including pancreatic, thyroid, salivary gland, breast, colorectal and lung, Roche said in its announcement this morning. However, diagnosing those types of tumors is difficult. Roche said that biomarker testing for NTRK gene fusions is the only way to identify people who may be eligible for treatment with Rozlytrek. The Swiss pharma giant is working with its subsidiary Foundation Medicine to develop personalized medicines and advanced diagnostics to help identify people with NTRK gene fusions using a companion diagnostic that is undergoing review, the company said.

Janet Freeman, co-founder of The ROS1ders, a group of patients and caregivers affected by ROS1-positive lung cancer, noted that approximately 40% of people with ROS1-positive non-small cell lung cancer have tumors that have spread to the brain. The approval of Rozlytrek means there is now a treatment option for the patients diagnosed with this rare type of cancer, Freeman said.

“The identification of actionable biomarkers like ROS1 has brought about significant progress in the treatment of lung cancer. This approval brings further hope to people with this rare type of the disease,” Freeman said in a statement.

Rozlytrek (entrectinib) is an oral medication for the treatment of locally advanced or metastatic solid tumors that harbor NTRK1/2/3. It is a selective tyrosine kinase inhibitor designed to inhibit the kinase activity of the TRK A/B/C and ROS1 proteins, whose activating fusions drive proliferation in certain types of cancer. Rozlytrek can block ROS1 and NTRK kinase activity and may result in the death of cancer cells with ROS1 or NTRK gene fusions, the company said.

Rozlytrek first won approval in Japan in June. The latest approval from the FDA was based on results from the integrated analysis of the pivotal Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA-372-001 trials, and data from the Phase I/II STARTRK-NG study. In ROS1-positive, metastatic NSCLC, Rozlytrek shrank tumors in 78% of people with disease, Genentech said in its announcement. Rozlytrek also shrank tumors in more than half of people with NTRK gene fusion-positive, locally advanced or metastatic solid tumors. Objective responses were observed across 10 tumor types. Objective responses to Rozlytrek were seen in people with central nervous system (CNS) metastases at baseline. In the STARTRK-NG study, Rozlytrek shrank tumors also in children and adolescents who had NTRK fusion-positive solid tumors including the patients with primary CNS tumors.

Sandra Horning, Roche’s chief medical officer and head of Global Product Development, said the FDA approval for the two rare types of cancer an important step that combines a targeted medicine and genomic testing.

Back to news