FDA approves drug that targets genetic features behind cancer cells
The Food and Drug Administration says it has approved the drug Vitrakvi for adult and pediatric patients whose cancers have a specific biomarker.
"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," FDA Commissioner Scott Gottlieb, M.D., said regarding . “We now have the ability to make sure that the right patients get the right treatment at the right time.”
Vitrakvi, the brand name of larotrectinib, has been given the FDA seal of approval for the treatment of adult and pediatric patients with solid tumors that contain a neurotrophic receptor tyrosine kinase gene fusion.
Researchers say the NTRK genes can become fused to other genes abnormally, which can result in the growth of tumors. While NTRK fusions are rare, they occur in cancers in many sites of the body, according to the FDA. Prior to Monday’s announcement, there had been no treatment for cancers that contain the mutation.
According to the FDA, larotrectinib demonstrated a 75 percent overall response rate to tested tumors. These responses were durable, with 73 percent of responses lasting at least six months, and 39 percent lasting a year or more at the time results were analyzed. Soft tissue sarcoma, salivary gland cancer, infantile fibrosarcoma, thyroid cancer and lung cancer tumors were among the tested groups that responded to the treatment.
Doctors warn, however, the drug doesn't work for all cancers — and it is expensive. The wholesale cost for the children's syrup is $11,000 for a month's supply and the oral capsules for adults wholesales for $32,000 per month. With insurance, most patients would pay $20 or less for a 30-day supply.
The success of the trials led to an accelerated approval from the FDA, allowing Vitrakvi to be used for patients with serious conditions that are currently unmet in regard to medicine.