According to a publication from CURE Today, the US Food and Drug Administration (FDA) recently approved Genentech, Inc’s Rozlytrek (generic name entrectinib) for the treatment of cancer patients who have specific genetic mutations. It’s only the third time ever that the regulatory agency has approved a cancer treatment targeting a specific genetic factor rather than a tumor itself.
The drug was approved in adult patients with non-small cell lung cancer with ROS1-positive and metastasized tumors, and also in patients older than 12 whose cancers have an “NTRK gene fusion.”
About the NTRK Gene Fusion
NTRK gene fusions involve any of three possible genes: NTRK1, NTRK2 or NTRK3. These genes are responsible for coding the production of neurotrophin receptors TRKA, TRKB and TRKC, respectively. These receptors interact with neurotrophins, molecules that play vitally important roles in the development and function of healthy neurons.
Fusion errors (a kind of genetic mutation) that occur in TRKA, TRKB, or TRKC are associated with increased risk of a multitude of adult and pediatric cancers. In many cases, early forms of these cancers can be effectively treated with TRK inhibitors, which can block the pro-cancer activity of mutated NTRK genes. However, in more advanced cases, cancers can become resilient to TRK inhibitors.
About Rozlytrek and Accelerated Approval
Rozlytrek is the first drug approved not only for NTRK fusions, but also for ROS1-positive patients with non-small cell lung cancer. The FDA based its decision to grant Rozlytrek accelerated approval based on data collected from four clinical studies of NTRK fusion patients, and one study of ROS1-positive patients.
In total, 54 patients with NTRK gene fusions participated in the studies. Those treated with Rozlytrek experienced significant improvement — 61% of those who responded to the drug experienced reductions in the size of their tumors for nine months or longer. In total, 7.4% of participants experienced “substantial reductions” in size or the complete disappearance of their tumors. 78% of ROS1-positive patients responded to treatment with 55% experiencing tumor shrinkage for periods lasting over a year. 5.9% had their tumors disappear entirely.
Though complete remission with treatment remains rare, the impressive clinical data begged action. The FDA granted the drug accelerated access on the basis of evidence that suggested the drug could potentially provide meaningful improvement on existing treatment options for NTRK fusion and ROS1-positive cancers.
Accelerated approval comes with the stipulation that Genentech must provide additional clinical data in the future. However, for the meantime, patients may access the drug as they would any drug under full market approval.
Because cancers are so diverse and complex, new treatments are few and far between. Tell Patient Worthy what you think of this exciting news!
