FDA Approves Rozlytrek

As more research is being done on cancer and its treatments, more targeted treatments are becoming available, particularly biologic drugs. The drug Rozlytrek (generic name entrectinib) was approved by the Food and Drug Administration (FDA) to treat patients 12 and older whose cancers have the genetic defect NTRK (neurotrophic tyrosine receptor kinase) gene fusion, and for whom there are no other effective treatments.1 This is significant because the drug is a cancer treatment that can treat different kinds of cancers that arise from different tissues (like breast cancer or lung cancer), as long as they share the common biomarker. These cancer drugs are often called “tissue agnostic.”1 The drug was also approved for the treatment of adult patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC).2

What is Rozlytrek?

Rozlytrek (generic name entrectinib) is a drug that is now FDA-approved to treat patients who are 12 years old and older who have solid tumors exhibiting an NTRK gene fusion and no alternative therapies are available.2 Drugs for pediatric populations are generally not explored until development has been underway for adults, along with approval and safety indications, but the efficacy of Rozlytrek for adolescent patients was based on adult data, along with safety data in 30 pediatric patients.2

Approval based on research

The drug was approved based on pooled data from the phase II trial STARTRK-2, the phase I STARTRK-1 trial, and the phase I ALKA-372-001 trial.2 Additional data was also taken from the phase I/II STARTRK-NG trial.2 Altogether, the trials were comprised of patients with 10 different tumor types and more than 19 histopathologies, including breast cancer, cholangiocarcinoma, gynecological cancer, colorectal cancer, pancreatic cancer, neuroendocrine cancer, NSCLC (non-small cell lung cancer), salivary gland cancer, sarcoma, and thyroid cancer.2 A fair amount of these patients also had central nervous system metastases at baseline.2

Overall, in patients with NTRK fusion-positive cancers, 57% had significant tumor shrinkage and overall response rate, and 7.4% had complete disappearance of the tumor.1 Among those with tumor shrinkage, the reduction lasted for 9 months or longer in 61% of patients, with the most common cancers included in this being lung, breast, salivary gland, thyroid, and colorectal.1 Among patients with metastatic NSCLC that is ROS1-positive, the overall response rate to Rozlytrek was 71%, with 5.9% having a complete disappearance of their cancer.1 For those with tumor shrinkage, 55% had the results lasting 12 months or more.1

Adverse reactions associated with taking Rozlytrek

Taking any medication is a risk, and any drug has the potential to cause adverse effects. Common adverse effects seen with Rozlytrek included fatigue, constipation, changes in taste, pain, anemia, cognitive disorders, vomiting, weight gain, increase in blood creatinine, vision disorders, shortness of breath, and myalgia.1,2 More serious side effects include congestive heart failure, central nervous system effects (depression, cognitive impairment, suicidal ideation), skeletal fractures, and liver damage.1 Women who are pregnant or breastfeeding should not take Rozlytrek, and those of reproductive age should use effective contraception while taking this medication, since it can cause harm to a fetus or baby.


For those with breast and lung cancers who are NTRK fusion-positive and those with ROS1-positive metastatic NSCLC, Rozlytrek might be a viable treatment option. Talk with your treatment team about the possible risks and benefits, and whether this drug may be right for you.

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