LL schreef op 6 februari 2023 12:42:
FDA Approves TAKHZYRO® to Prevent HAE Attacks in Children 2 Years of Age and OlderThe U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for the expanded use of Takeda‘s TAKHZYRO® (lanadelumab-flyo) for prophylaxis to prevent attacks of HAE in pediatric patients 2 to <12 years of age. Prior to the 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.
“The approval for TAKHZYRO in pediatric patients as young as 2 years of age brings a welcome and important addition to treatment options available for children living with HAE,” says Anthony Castaldo, President and CEO of the U.S. Hereditary Angioedema Association (HAEA).
The sBLA approval was supported by extrapolation of efficacy data from the HELP Study, a Phase 3 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 3 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.
“Today’s approval of the expanded indication of TAKHZYRO represents a significant step forward for the HAE community as it helps some of its youngest patients who are living with the disease to have a long-term prophylaxis treatment available to them,” says Julie Kim, President, U.S. Business Unit and U.S. country head at Takeda. “Takeda is a committed leader in the rare disease space, and today’s approval underscores our confidence in TAKHZYRO, as well as our dedication to addressing the needs of HAE patients through continued research, clinical programs and real-world data collection.”
TAKHZYRO was originally approved in the U.S. in 2018 for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years and older.1 It is currently available in more than 60 countries around the world and is supported by a robust clinical development program, which includes one of the largest prevention studies in HAE with the longest active treatment duration.
(Source: Takeda)