Unlike other treatments that focus on a specific form of cancer (breast, kidney, liver etc.), Vitrakvi (larotrectinib) uses a different approach and targets neurotrophic receptor tyrosine kinase (NTRK) gene fusions within the mutated cells. This allows the drug to have clinical benefits over numerous unique tumor types, including lung, thyroid, melanoma, GIST, colon, soft tissue sarcoma, salivary gland and infantile fibrosarcoma.
“The FDA approval of larotrectinib marks an important milestone in how we treat cancers that have an NTRK gene fusion—a rare driver of cancer. I have seen firsthand how treatment with larotrectinib, which is designed specifically for this oncogenic driver, can deliver clinically meaningful responses in patients with TRK fusion cancer, regardless of patient age or tumor type”, said David Hyman, M.D., chief of the Early Drug Development Service at Memorial Sloan Kettering Cancer Center and lead trial investigator.
Vitrakvi has the potential to address solid tumors that are either metastatic or where surgical intervention could not yield satisfactory outcomes. This is only the second ever tissue-agnostic FDA approved therapy for cancer.
The decision was based on three multicenter, open-label, single-arm clinical trials that gathered 55 patients, 12 of which under the age of 18. A wide number of conditions were presented, the most common being salivary gland tumors (22%), soft tissue sarcoma (20%), infantile fibrosarcoma (13%) and thyroid cancer (9%).
OThe overallresponse rate was 75%, including 22% complete responses and 53% partial responses. Median duration of response was not yet reached, with 73% of cases showing a response for more than six months.
“We’re seeing scientific advancements, like genomic testing capable of detecting an NTRK gene fusion, beginning to transform the treatment of cancer and provide new options for patients“, noted in the same press release Andrea Stern Ferris, president and chief executive officer of the LUNGevity Foundation.