Bayer/Loxo drug approved in cancers with certain mutation

The FDA has approved Bayer and Loxo’s Vitrakvi (larotrectinib) for adults and children whose cancers have a specific genetic feature, or biomarker.

This is only the second time the agency has approved a cancer treatment on the basis of its efficacy against a specific biomarker, rather than the location in the body in which the tumour originated.

Vitrakvi is now approved for adult and paediatric patients with solid tumours that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation.

Tumours must be metastatic or where surgical resection is likely to result in severe morbidity, and where no satisfactory alternatives are available.

The FDA said the approval marks a new paradigm in the development of cancer drugs that are “tissue agnostic”.

The regulator has developed a guidance document for such drugs, which was released earlier this year.

FDA Commissioner Scott Gottlieb said: “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.”

“This new site-agnostic oncology therapy isn’t specific to a cancer arising in a particular body organ, such as breast or colon cancer. 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.”

Robert LaCaze, member of the executive committee of Bayer’s Pharmaceuticals Division and head of its Oncology Strategic Business Unit, said: “Today's approval of Vitrakvi is the culmination of years of hard work and research by many people to bring the first ever treatment to patients with TRK fusion cancer.

“TRK fusions are rare, but occur across many different tumour types. In this era of precision medicine, we are delivering on Bayer’s commitment to advance the future of cancer care while providing value for patients and physicians.”

The first cancer drug approved on the basis of a biomarker was Merck & Co’s Keytruda (pembrolizumab).

Keytruda was FDA approved in May last year for unresectable or metastatic tumours with an instability-high or mismatch repair deficient biomarker, adding to a long list of other uses.