Approval Announcement
WHIPPANY, N.J.--(BUSINESS WIRE) April 10, 2025 --Bayer today announced that the U.S. Food and Drug Administration (FDA) has granted full approval for Vitrakvi® (larotrectinib), a first-in-class TRK inhibitor.
Approved Indication
Vitrakvi is indicated for the treatment of adult and pediatric patients with solid tumors that meet the following criteria:
Harbor a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation
Are metastatic, or where surgical resection is likely to result in severe morbidity
Have no satisfactory alternative treatments, or have progressed following prior treatment
Vitrakvi has demonstrated clinically meaningful and durable responses across a range of NTRK fusion-positive solid tumors. It was first granted accelerated approval by the FDA in November 2018.
Executive Comments
Bayer Leadership Statement
“This first full approval of an NTRK inhibitor by the FDA represents the culmination of research and dedication by the Bayer team,” said Chandra Goda, Executive Director, U.S. Vitrakvi Brand Lead. “We are proud to deliver on our promise for patients with this significant step forward, providing a treatment option for pediatric and adult patients living with NTRK gene fusion-positive cancers. This milestone reinforces Bayer's commitment to delivering innovative solutions that address the unique needs of patients and their families.”
Patient Advocacy Statement
"The full approval of Vitrakvi by the FDA is a welcome step forward, solidifying its place as a treatment option for patients with NTRK gene fusion-positive cancers," said Andrea Ferris, President and CEO, LUNGevity Foundation. "This milestone not only benefits patients today but also paves the way for further advancements in NTRK gene therapies in the future."
Basis for Approval: Clinical Trial Data
The approval is based on data from three multicenter, open-label, single-arm clinical trials:
LOXO-TRK-14001 (NCT02122913)
SCOUT (NCT02637687)
NAVIGATE (NCT02576431)
Study Population
The analysis included 339 pediatric and adult patients with unresectable or metastatic solid tumors harboring an NTRK gene fusion. All patients had progressed following systemic therapy (if available) or would require surgery with significant morbidity for locally advanced disease.
Efficacy Endpoints
Primary efficacy outcomes were overall response rate (ORR) and duration of response (DOR), assessed by a blinded independent review committee (BIRC) per RECIST v1.1. Safety was evaluated in 444 patients across the three trials.
Pooled Efficacy Results
ORR: 60% (95% CI: 55%–65%)
Complete response (CR): 24% (including 5% pathological complete response, defined as no viable tumor cells and negative margins post-resection with no other disease sites)
Partial response (PR): 36%
Median DOR: 43.3 months (95% CI: 32.5–NE (not evaluable))
Safety Profile
Most common (≥20%) adverse reactions (including laboratory abnormalities):Increased AST, increased ALT, anemia, hypoalbuminemia, musculoskeletal pain, increased alkaline phosphatase, leukopenia, lymphopenia, neutropenia, hypocalcemia, fatigue, vomiting, cough, constipation, pyrexia, diarrhea, nausea, abdominal pain, dizziness, and rash.
Serious adverse events (AEs) included central nervous system (CNS) problems, bone fractures, and liver problems.
About Vitrakvi® (larotrectinib)
Vitrakvi is a first-in-class oral TRK inhibitor, exclusively designed to inhibit the TRK family of proteins (TRKA, TRKB, TRKC).
Mechanism of Action
In in vitro and in vivo tumor models, Vitrakvi demonstrates anti-tumor activity in cells with constitutive activation of TRK proteins (resulting from gene fusions, deletion of regulatory domains, or overexpression). Constitutively activated chimeric TRK fusion proteins act as oncogenic drivers, promoting tumor cell proliferation and survival.
