FDA Approves Augtyro for Children, Adults With NTRK-Positive Solid Cancers

The FDA approved Augtyro for some with NTRK-positive solid tumors.

The Food and Drug Administration (FDA) granted an accelerated approval to Augtyro (repotrectinib) for adults and children aged 12 years or older with solid tumors that harbor an NTRK gene fusion.

More specifically, the approval is for patients whose disease is locally advanced (grew beyond the initial location, but not to other body parts) or metastatic (spread to other body parts) where surgical removal of the cancer is likely to result in severe morbidity or health complications. Patients had to have undergone at least prior treatments and have no other satisfactory therapies available to be eligible for Augtyro, the FDA explained in a press release.

“NTRK fusion-positive tumors can present challenges in the clinical setting, which is why it is important that we have additional treatment options for these patients,” said Dr. Alexander Drilon, TRIDENT-1 global trial lead and Chief of the Early Drug Development Service at Memorial Sloan Kettering Cancer Center, in a press release issued by Bristol Myers Squibb, the manufacturer of Augtyro. “The FDA approval of [Augtyro] adds an important tool to our toolbox, offering oncologists a next-generation TKI that can be used across a broad range of NTRK fusion-positive solid tumors for both TKI-naïve and TKI-pretreated patients.”

The approval is based on findings from the TRIDENT-1 clinical trial, which included 88 adults with locally advanced or metastatic NTRK gene fusion-positive solid tumors that progressed (got worse) on either a prior tyrosine kinase inhibitor (TKI; 48 patients) or who did not previously receive a TKI (40 patients). The study did not include patients who had symptomatic brain metastases.

Researchers on the study assessed patients every eight weeks. The main goal of the trial was to measure the overall response rate (ORR; percentage of patients whose cancer shrinks or disappears from treatment) and duration of response (DOR; time patients live on treatment without their disease spreading), as determined by a group of independent experts who are not affiliated with the study.

Findings showed that the confirmed ORR in the group of patients who previously received a TKI drug was 50%, and it was 58% in patients who did not previously receive a TKI. The median DOR was 9.9 months in the TKI-pretreated group and not estimable in the TKI-naïve group. When a data point is not estimable, that means that not enough study participants experienced an outcome (in this case disease growth or spreading after treatment response) for the researchers to calculate an average.

Augtyro is a drug that is taken in the form of a pill. It was previously approved for patients with advanced or metastatic ROS1-positive non-small cell lung cancer. The drug is a TKI that works by targeting and blocking the NTRK or ROS1 genes, which could play a role in the development and growth of cancerous cells.

The most common side effects that occurred in 20% or more of patients was dizziness, foul taste, peripheral neuropathy (weakness and numbness in hands and feet), constipation, difficulty breathing, difficulty coordinating body movements (ataxia), cognitive impairment, muscular weakness and nausea.

“Cancer can be frightening regardless of the type, but having a rare gene fusion driving it can be especially stressful and isolating,” Susan Spinosa, president and patient co-founder of NTRKers, a patient advocacy group, said in the press release. “It’s exciting to know that there’s a new targeted therapy option for patients with NTRK-positive gene fusions, as this may offer hope to patients and their loved ones navigating this difficult journey.”

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