FDA approves Vitrakvi for solid tumors driven by rare mutation

  • US Food and Drug Administration

  • curated by Yael Waknine
  • Clinical Essentials
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  • FDA has granted accelerated approval for larotrectinib (Vitrakvi), to treat solid tumors that have a targeted biomarker — neurotrophic receptor tyrosine kinase (NTRK) gene fusion — without a known acquired resistance mutation.
  • Larotrectinib capsules and oral solution are indicated for:
    • Patients with metastatic disease, or where surgical resection is likely to yield severe morbidity, and
    • have no satisfactory alternatives or that have progressed following treatment.

Why this matters

  • First “site agnostic” therapy specifically developed to target a common biomarker.
  • See also: FDA greenlights first cancer treatment for any solid tumor sharing a specific biomarker

Clinical study designs

  • 3 single-arm, open-label clinical trials involving 55 adult and pediatric patients (median age, 45 years; range, 4 months to 76 years).
  • Salivary gland tumors (22%), soft tissue sarcoma (20%), infantile fibrosarcoma (13%), and thyroid cancer (9%) were most common.
  • Funding: Bayer.  

Key results

  • Objective response rate, 75% (95% CI, 61%-85%).
  • Complete response rate, 22%.
  • Duration of response (DOR): ≥6 months, 73%; ≥9 months, 63%; ≥12 months, 39%.
  • Median DOR (range, 1.6+, 33.2+) and PFS had not been reached at time of analysis.

Adverse events

  • Common adverse reactions (≥20%) were fatigue, nausea, dizziness, vomiting, increased liver enzymes, constipation, and diarrhea.
  • Labeling carries warnings regarding risks for neurotoxicity, hepatotoxicity, and embryo-fetal toxicity.

Prescribing Information, click  here.

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