News: FDA Approves “Precision Drug” That Treats Multiple Cancers

News: FDA Approves “Precision Drug” That Treats Multiple Cancers

The drug targets a rare genetic mutation that can be found in a variety of cancers.

The U.S. Food and Drug Administration (FDA) has approved a new cancer drug designed to fight a specific genetic mutation in tumors regardless of where they grow in the body.

The drug, called Vitrakvi (larotrectinib), doesn’t target just one type of cancer, such as breast or colon cancer. Instead, it treats tumors that carry a certain genetic feature, or biomarker, known as neurotrophic receptor tyrosine kinase (NTRK) fusion.

When they function normally, NTRK genes lead to the formation in the body of a type of protein called TRK proteins. But in abnormal cases, NTRK genes may become fused to other unrelated genes, which could trigger the growth of tumors. These mutations are uncommon, but they can occur in many cases of a very rare cancer affecting children, called infantile fibrosarcoma, as well as a small percentage of solid tumors that arise in various parts of the body, including the lungs, thyroid gland and colon.

Vitrakvi, developed by Loxo Oncology, was approved under the FDA’s accelerated approval program, which enables the agency to “green light” drugs for serious conditions in order to fill an unmet medical need based on promising initial clinical trials.

The drug was studied in three trials, involving 55 children and adult patients diagnosed with solid tumors with NTRK gene fusion that had spread and did not respond to any other treatments. These patients’ tumors could also not be surgically removed safely.

Overall, the drug had a 75 percent overall response rate. Of the 55 patients included in the trial, 53 percent had a partial response, in which they experienced either a reduction in the size of their tumors or tumors that stopped growing entirely. Meanwhile, 22 percent had a complete response, or their cancer was no longer detectable. Of these responses, 73 percent lasted for at least six months while 39 percent lasted for a year or more, the FDA reported.

Common side effects of the drug include fatigue, nausea, cough, constipation, diarrhea, dizziness, vomiting and liver damage. The drug should not be taken by pregnant or breastfeeding women because it could harm a developing fetus or newborn.

The new cancer drug targets a rare mutation and more clinical trials are needed to confirm Vitrakvi’s long-term safety and effectiveness, but this milestone is important for a couple of reasons. For one, it’s a significant step forward for precision medicine, which challenges a one-size-fits-all approach to cancer treatment. What’s more, this single drug could help treat many tumor types.

"Today’s approval marks another step in an important shift toward treating cancers based on their tumor genetics rather than their site of origin in the body," said FDA Commissioner Scott Gottlieb, MD, in a news release. “Its approval reflects advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine. We now have the ability to make sure that the right patients get the right treatment at the right time.”

The population of people affected by NTRK fusion is also small, but for these people, the benefits could be dramatic. Until now, there were no drugs for certain cancers that often carry this mutation. Loxo Oncology says Vitrakvi is now available as an oral capsule or a liquid formulation for the treatment of both adults and children.

The FDA has more information on its approval of Vitrakvi.

Medically reviewed in November 2018.

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