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FDA approves second tissue-agnostic cancer drug

Bayer and Loxo’s treatment carries a hefty price tag

by Lisa M. Jarvis
November 28, 2018 | A version of this story appeared in Volume 96, Issue 48


The molecular structure of Loxo and Bayer's TRK fusion inhibitor Vitrakvi.

The US Food and Drug Administration has approved another cancer drug prescribed based on the genetic profile, rather than the location, of a person’s tumor. Vitrakvi, from Bayer and Loxo Oncology, treats the roughly 1% of cancer patients whose tumors are caused by a chromosomal mismatch called TRK gene fusions.

Vitrakvi is only the second drug to reach the market with a so-called “tissue agnostic” approval. In May 2017, Merck & Co.’s immunotherapy Keytruda won FDA’s blessing to treat anyone with a defect in mismatch repair genes, although the drug had previously been approved for melanoma and lung, head, and neck cancers.

Both drugs have proven remarkably effective in patients with the right genetic markers, and they were developed at breakneck speed. Loxo enrolled the first patient—a 42-year-old woman who had run out of treatment options for her sarcoma—in a study of Vitrakvi in March 2015. By 2017, the firm had shown that the drug shrunk tumors in 76% of people with TRK gene fusions across many kinds of cancers. That data prompted Bayer to pay Loxo upwards of $1.5 billion to jointly develop Vitrakvi and a follow-on compound, LOXO-195.

The partners are charging dearly for that innovation: The annual wholesale price of Vitrakvi is $393,600 for pills or $132,000 for a liquid for children and adults who can’t take pills. Bayer has a plan to give insurance companies and patients their money back if the drug shows no benefit after 90 days of treatment.

The companies’ next challenge is finding patients for Vitrakvi. TRK gene fusions are so rare that oncologists don’t often look for them. Still, Leerink Partners forecasts Vitrakvi will reap $700 million in annual US sales by 2030.

And while oncologists have been frustrated at the cost of new cancer drugs, some see Vitrakvi’s efficacy as a differentiator. “There is nothing more expensive than drugs that don’t work,” says Razelle Kurzrock, head of the Center for Personalized Cancer Therapy at the University of California, San Diego’s Moores Cancer Center. “This is a very expensive drug that works.”

She calls the high price a strong incentive for others to develop effective drugs for rare cancers.


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