On Monday, the United States Food and Drug Administration (FDA) announced the accelerated approval of Vitrakvi (larotrectinib), a treatment for patients whose cancers have a specific genetic feature (biomarker). Vitrakvi targets the NTRK gene.
This is the second time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated. The approval marks a new paradigm in the development of cancer drugs that are “tissue agnostic.” It follows the policies that the FDA developed in a guidance document released earlier this year.
FDA Commissioner Scott Gottlieb, M.D. states:
"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, M.D. "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. This type of drug development program, which enrolled patients with different tumors but a common gene mutation, wouldn’t have been possible a decade ago because we knew a lot less about such cancer mutations. Using our breakthrough therapy designation and accelerated approval processes, we support innovation in precision oncology drug development and the evolution of more targeted and effective treatments for cancer patients. This is especially true when it comes to pediatric cancers. We’re committed to continuing to advance a more modern framework of clinical trial designs that support more targeted innovations across disease types based on our growing understanding of the underlying biology of diseases like cancer."
To read the full FDA press release, click here.
Vitrakvi is marketed by Bayer and will be made available to adults and children who have the NTRK gene. Eligibility can depend upon whether the cancer has spread (metastasized), whether it responded to traditional/alternative treatments, and/or whether it returned and progressed following treatments.
Overall, this type of cancer is rare, but can occur across a broad range of tumor types, including cancers of the salivary gland, thyroid, lung, colon, appendix, breast and pancreas.
Bayer also states:
“Every patient who needs the life-saving medication will have affordable access.”
That’s very important, as the wholesale price of a 30-day supply of Vitrakvi is $32,800 for adults.
Forbes reports:
The company says monthly out-of-pocket costs for the majority of patients will be $20 or less. "Bayer will ensure that no eligible patient with TRK fusion cancer will go without this highly effective therapy," the company said in a statement...The company will help patients pay expensive co-pays, and will provide Vitrakvi for free while insurance details are worked out. If a patient can't afford the medicine, a charity funded by Bayer will provide the drug at no cost. The drug firm promises that if patients don't show a clinical benefit in the first three months of treatment, it will refund the money spent by insurers or
Though many of us may not see cancer cured in our lifetimes, it’s exciting to see important advances and good news like this taking place every year. As for Bayer, the company has had its share of good and bad press over the decades. Let’s hope in this case, other pharmaceuticals will follow Bayer’s lead.