FDA Grants Accelerated Approval for New Cancer Drug Repotrectinib

FDA

WASHINGTON, D.C. — On Thursday, the Food and Drug Administration granted accelerated approval to repotrectinib (AUGTYRO), a new drug for treating certain advanced solid tumors in patients aged 12 and older. This approval covers tumors with a specific genetic feature known as neurotrophic tyrosine receptor kinase (NTRK) gene fusion.

Repotrectinib is designed for patients whose cancer is locally advanced or metastatic and who have either worsened after treatment or lack satisfactory alternative therapies. The drug was evaluated in a clinical trial called TRIDENT-1, involving 88 adult patients with advanced NTRK gene fusion-positive tumors.

The trial included two groups: one that had been treated with a previous TRK tyrosine kinase inhibitor (TKI) and another that had not. In the TKI-naïve group, the overall response rate (ORR) was 58%, meaning more than half of the patients saw their tumors shrink. In the TKI-pretreated group, the ORR was 50%. The duration of response varied, with the TKI-naïve group showing results that were not yet fully measurable, while the TKI-pretreated group had a median response duration of nearly 10 months.

Patients in the trial were regularly assessed for tumor progression every eight weeks. Those with symptomatic brain metastases were excluded from the study.

Common side effects of repotrectinib include dizziness, altered taste, peripheral neuropathy (nerve damage), constipation, difficulty breathing, fatigue, ataxia (lack of muscle control), cognitive impairment, muscle weakness, and nausea. These side effects occurred in more than 20% of patients.

The recommended dosage for repotrectinib starts at 160 mg taken orally once daily for the first 14 days. After this initial period, the dose increases to 160 mg twice daily, which can be taken with or without food. Treatment continues until the disease progresses or the patient experiences unacceptable toxicity.

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This accelerated approval means that repotrectinib can be made available to patients sooner, based on preliminary evidence showing it works against tumors with NTRK gene fusions. The approval is conditional on further verification and description of clinical benefits in additional trials.

The development of repotrectinib offers new hope for patients with challenging forms of cancer, providing a targeted therapy option where few existed before.

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