WHIPPANY, N.J.— The U.S. Food and Drug Administration (FDA) has granted full approval for Bayer’s VITRAKVI® (larotrectinib), marking a significant milestone in the treatment of solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion in both adult and pediatric patients.
The decision follows the initial accelerated approval granted in 2018 and is based on results from three clinical trials demonstrating the drug’s efficacy and safety. VITRAKVI is now fully approved for patients whose tumors are metastatic or cannot be surgically removed without severe consequences, and who have no satisfactory treatment alternatives or whose disease has progressed despite prior treatment.
VITRAKVI is the first therapy specifically developed to target TRK fusion proteins, which drive cancer growth in a range of tumors. According to Bayer, the treatment has shown consistent, durable responses across multiple tumor types in both children and adults.
“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 and U.S. Brand Lead for VITRAKVI. “This milestone reinforces Bayer’s commitment to delivering innovative solutions that address the unique needs of patients and their families.”
The approval was supported by data from three open-label clinical studies—LOXO-TRK-14001, SCOUT, and NAVIGATE—encompassing 339 patients with unresectable or metastatic NTRK fusion-positive tumors. The pooled data revealed an overall response rate (ORR) of 60%, including a 24% complete response rate. The median duration of response was over 43 months.
Andrea Ferris, President and CEO of the LUNGevity Foundation, called the approval “a welcome step forward,” emphasizing its importance not only for current patients but also for the future of targeted cancer therapy.
While VITRAKVI has shown promising results, the treatment comes with potential side effects. Common adverse reactions included fatigue, nausea, vomiting, and liver enzyme abnormalities. More serious events reported involved central nervous system effects, bone fractures, and liver issues.
With this full FDA approval, VITRAKVI now stands as a validated treatment option for patients with NTRK gene fusion-positive cancers, further advancing precision oncology.
