FDA expands pediatric solid tumor indication for Rozlytrek, approves new oral formulation
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The FDA granted accelerated approval to entrectinib for infants aged 1 month and older who have solid tumors that harbor neurotrophic tyrosine receptor kinase, or NTRK, gene fusion for whom there are no effective treatments.
The indication includes individuals with metastatic disease for whom surgical resection would likely result in severe morbidity or those who are at high risk for disease progression after treatment.
FDA previously granted accelerated approval to entrectinib (Rozlytrek, Genentech) — a kinase inhibitor — for pediatric patients 12 years of age and older for the same indication. The treatment is also indicated for adults with ROS1-positive metastatic non-small cell lung cancer as detected by an FDA-approved test.
The regulator has also approved a new oral pellet formulation for the agent, with information on how to make an oral suspension from capsules included in the prescribing information.
“This indication is approved under accelerated approval based on overall response rate and duration of response,” the FDA said in a press release. “Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.”
The FDA approved the expanded indication based on pooled results from pediatric patients with NTRK-positive solid tumors who received entrectinib as part of two multicenter single-arm clinical trials — STARTRK-NG and TAPISTRY. Overall response rate as determined by blinded independent central review served as the primary efficacy outcome for both trials.
Investigators reported an ORR of 70% (95% CI, 51-84) among 33 pediatric patients and median DOR of 25.4 months (95% CI, 14.3 to not evaluable).
A pooled safety analysis of 76 pediatric patients showed the most frequent treatment-related adverse events in greater than 20% of patients included pyrexia, constipation, weight gain, vomiting, diarrhea, nausea, cough, fatigue, pain in extremity, skeletal fracture, decreased appetite, headache, abdominal pain, urinary tract infection, upper respiratory tract infection and nasal congestion.
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