FDA Expands Rozlytrek Indication to Include Some Solid Tumors in Children at Least 1 Month Old


This expanded indication broadens the use of Rozlytrek for solid tumors to include children one month old, with the original approval in 2019 only including children one year and older.

The Food and Drug Administration (FDA) approved an expanded indication for Rozlytrek (entrectinib) to include children older than one month with solid tumors that harbor the NTRK gene fusion without a known acquired resistance mutation, have metastatic disease or where surgical removal may result in severe morbidity and whose disease has progressed after treatment.

The FDA also approved a new oral pellet formulation of Rozlytrek, according to the statement from the agency.

Rozlyrek was initially approved by the FDA in 2019 for the treatment of patients aged 12 years and older.

The efficacy of Rozlytrek in NTRK-positive tumors was assessed in children from two different clinical trials: STARTRK-NG and TAPISTRY. In particular, 33 children received Rozlytrek in a dose based on body surface area, which could range anywhere between 20 milligrams to 600 milligrams orally or through an enteral feeding tube once per day.

Several factors were assessed throughout these trials including overall response rate (the percentage of patients who achieved a partial or complete response to treatment) and duration of response (the time from when a patient responded to treatment until disease progression or death).

In the 33 patients included in this analysis, the overall response rate was 70% with a median duration of response of 25.4 months. Of note, the most common cancers in this group of patients were infantile fibrosarcoma, which is the most common soft tissue sarcoma in children younger than 1 year old, and primary central nervous system tumors.

In another analysis of 76 children treated with Rozlytrek, the most common side effects, occurring in at least 20% of patients, included constipation, fever, vomiting, increased weight, nausea, diarrhea, fatigue, cough, skeletal fracture, pain in the extremity, headache, decreased appetite, urinary tract infection, abdominal pain, nasal congestion and upper respiratory tract infection.

When Rozlytrek was originally approved in August 2019, it was for the treatment of adults and adolescents aged 12 years and older whose cancers have the NTRK gene fusion and for adults with non-small cell lung cancer whose disease is ROS1-positive and has metastasized.

Before the drug was approved in 2019, the FDA noted that there were no effective treatments for patients with NTRK gene fusions.

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