This week, the U.S. Food and Drug Administration granted accelerated approval to larotrectinib (Vitrakvi®), a treatment for adult and pediatric patients whose cancers have a specific genetic alteration, called a NTRK fusion.
Here are the most important things you should know about this new therapy and what it means for our patients:
- The approval marks a new paradigm in the development of cancer drugs that are “tissue agnostic” – which means that we can help treat the individual by what is driving the cancer rather than where the cancer originated in the body. This is the second time that the FDA has approved a drug for a cancer in a tissue agnostic setting – the first being, pembrolizumab (KEYTRUDA®) for MSI-High tumors (MSI-H mutations result when genes that regulate DNA don’t work correctly).
- Prior to larotrectinib’s approval, there had been no treatment for cancers that express this alteration.
- It is an oral therapy, approved in liquid form for children and in a pill form for adults, twice daily. Side effects are largely manageable (gastrointestinal, liver enzyme elevations, fatigue) helping these patients remain on extended therapy.
- From the initial studies, it has been observed that 3 out of 4 patients have seen their cancer shrink substantially; and 1 in 5 patients have had complete remissions. Meaningfully, 40% of study participants have been responding for a full year, and some patients are even approaching 2 years.
- Sarah Cannon participated in this groundbreaking study and the approval reinforces our position on the importance of molecular profiling. By better understanding the potential genetic drivers of cancer, we are able to investigate better targeted approaches. Testing and targeted drug development can advance our ability to get the right therapy to the right patient at the right time.
Dr. Spigel, Sarah Cannon’s Chief Scientific Officer, discussed this approval Wednesday on HLN News. Watch the clip here.