Second Ever Cancer Treatment Based on Biomarkers Approved by FDA

On November 26, 2018, a new drug was given accelerated approval by the FDA. Not only that, but it was granted orphan drug designation and breakthrough therapy designation. The drug was designed to treat adult and pediatric cancer patients but it’s unique because it isn’t for one particular kind of tumor. It works according to bio-marker, as opposed to tumor location.

The Treatment

The drug is called Vitrakyi and it is only the 2nd cancer treatment approved by the FDA which is based on biomarkers. Specifically, this drug was created for patients who have a neurotrophic receptor tyrosine kinase (NTRK) gene. It was developed to treat patients who-|

  • are metastatic
  • have progressed after treatment
  • have no alternative treatment option
  • are not expected to respond well to surgery

FDA Commissioner Scott Gottleib emphasizes that this kind of approval was not even fathomable a decade ago. But with our continuously increasing understanding of cancer mutations, we are able to design more therapies which focus on common genes. These therapies have shown to be incredibly effective as they are more targeted than traditional approaches.

Some cancers which may be affected by this new approval are infantile fibrosarcoma, mesoblastic nephroma (cellular or mixed), thyroid cancer, lung cancer, salivary gland cancer, soft tissue sarcoma, and mammary analogue secretory carcinoma. Some of these cancers had no approved treatment before this approval.

The Clinical Trials

There were three clinical trials for Vitrakvi prior to its approval. These included adult and pediatric patients with a NTRK gene fusion. There were 55 participants in total. For 75%, the drug was responsive. The response lasted 6 months in 73% of these patients and one year in 39% of patients.

These results are extremely promising. That said, further clinical trials are currently being planned to confirm its potential. Vitrakvi’s accelerated approval means the FDA saw the unmet medical need it could fill for cancer patients and issued approval based on a surrogate endpoint. But more research on this treatment is still needed.

Stay tuned to hear more updates on Vitrakvi! In the meantime, you can read more about its upcoming clinical trials and its current designations here.


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