USFDA Expands Anti Cancer Drug Entrectinib Indication For Pediatric Patients
On Friday the United States Food and Drug Administration granted accelerated approval to entrectinib an anti-cancer drug sold under the brand name Rozlytrek - used to treat non-small cell lung cancer and solid tumors for pediatric patients older than 1 month.
The drug was approved for pediatric patients with solid tumours as a second-line treatment where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory standard therapy. This application was granted priority review, breakthrough designation and orphan drug designation.
In August 2019, the FDA granted accelerated approval to entrectinib for pediatric patients of 12 years of age and older for this indication. FDA also approved a new oral pellet formulation for entrectinib, and the prescribing information now includes instructions for making an oral suspension from the capsules.
Efficacy in neurotrophic tyrosine receptor kinase (NTRK)-positive tumours was investigated in 33 pediatric patients who received the drug based on body surface area in one of two multicenter, single-arm clinical trials: STARTRK-NG or TAPISTRY.
Among the 33 pediatric patients, the (overall response rate) ORR was 70 per cent and median duration of response (DOR) was 25.4 months. The most common cancers were primary central nervous system tumours and infantile fibrosarcoma.
In the pooled safety population of pediatric patients receiving entrectinib (n=76), the most common adverse reactions were pyrexia, constipation, increased weight, 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.
This indication is approved under accelerated approval based on the overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
