FDA approves sBLA for Takhzyro in pediatric patients with hereditary angioedema.- Takeda

Prior to approval, the only approved routine prophylaxis treatment options for children 6 to><12 years of age required dosing every three to four days, and children with hae 2 to><6 years of age had no approved prophylaxis treatment, making takhzyro the first prophylaxis treatment for this age group. the recommended dose is 150 mg 1 ml solution in a single-dose prefilled syringe every four weeks in patients 2 to><6 years of age and every two weeks in patients 6 to><12 years of age.></12></6></6></12></12>

The sBLA approval was supported by extrapolation of efficacy data from the HELP Study, a Phase III trial that included patients 12 to <18 years of age, and additional pharmacokinetic analyses showing similar drug exposures between adults and pediatric patients, as well as safety and pharmacodynamic data from the spring study, an open-label phase iii trial in 21 hae patients 2 to><12 years of age. the primary objectives of the spring study were the safety and pharmacokinetics of takhzyro. the most common treatment-related treatment emergent adverse events in the study were injection site pain (29%), injection site erythema (14%), injection site swelling (5%), administration site pain (5%) and injection site reaction (5%). the prevention of hae attacks was measured as a secondary objective. takhzyro reduced the rate of hae attacks in pediatric patients by a mean of 94.8% compared to baseline, from 1.84 attacks per month to 0.08 attacks during the 52-week treatment period (n="21)." the majority of patients (76.2%, n="16)" were attack-free with an average of 99.5% attack-free days. these efficacy results are from an open-label, non-controlled trial, and the study was not designed for statistical hypothesis testing. further confirmatory studies are required to draw any conclusions from these data.></12></18>