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  6. FDA grants accelerated approval to repotrectinib for adult and pediatric patients with NTRK gene fusion-positive solid tumors
  1. Resources for Information | Approved Drugs

FDA grants accelerated approval to repotrectinib for adult and pediatric patients with NTRK gene fusion-positive solid tumors

On June 13, 2024, the Food and Drug Administration granted accelerated approval to repotrectinib (AUGTYRO, Bristol-Myers Squibb Company) for adult and pediatric patients 12 years and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and that have progressed following treatment or have no satisfactory alternative therapy.

Full prescribing information for Augtyro will be posted on Drugs@FDA.

Efficacy and Safety

Efficacy was evaluated in TRIDENT-1 (NCT03093116), a multicenter, single-arm, open-label, multi-cohort trial in 88 adult patients with locally advanced or metastatic NTRK gene fusion-positive solid tumors who had either received a prior TRK tyrosine kinase inhibitor (TKI) (n=48) or were TKI-naïve (n=40). All patients were assessed for central nervous (CNS) lesions at baseline, and patients with symptomatic brain metastases were excluded. Tumor assessments were performed every 8 weeks.

The major efficacy outcome measures were overall response rate (ORR) and duration of response (DOR) according to RECIST v1.1 as assessed by blinded independent central review. Confirmed ORR in the TKI-naïve group was 58% (95% [confidence internal] CI: 41, 73) and 50% (95% CI: 35, 65) in the TKI-pretreated group. Median DOR was not estimable (NE) (95% CI: NE, NE) in the TKI-naïve group and 9.9 months (95% CI: 7.4, 13.0) in the TKI-pretreated group.

The most common (>20%) adverse reactions were dizziness, dysgeusia, peripheral neuropathy, constipation, dyspnea, fatigue, ataxia, cognitive impairment, muscular weakness, and nausea.

The recommended repotrectinib dose is 160 mg orally once daily for 14 days, then increased to 160 mg twice daily with or without food, until disease progression or unacceptable toxicity.

Expedited Programs

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

This application was granted priority review, fast track designation, breakthrough therapy designation, and orphan drug designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

Follow the Oncology Center of Excellence on X (formerly Twitter) @FDAOncology.

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