TAKHZYRO is a completely human monoclonal antibody that particularly binds and reduces plasma kallikrein activity.
Before this approval, the only approved routine prophylaxis treatment options for children from 6 to less than 12 years of age was dosing every three to four days, while children with HAE in the age group of 2 to less than 6 years had no approved prophylaxis treatment, thereby making TAKHZYRO the first prophylaxis treatment for this age group.
The dose recommended is 150 mg/1 mL solution in a single-dose prefilled syringe every four weeks in patients aged from two to less than 6 years of age and every two weeks in patients from six to less than 12 years of age.
HAE attacks can involve serious and debilitating swelling in the abdomen, face, feet, genitals, hands and throat.
The sBLA approval was backed by extrapolation of efficacy data from the HELP Study, a Phase III trial that included patients 12 to less than 18 years of age, and further pharmacokinetic analyses indicating 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 from 2 to less than 12 years of age.
Takeda US Business Unit and US country head Julie Kim said: “The 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.
“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.”
Originally, TAKHZYRO received approval in the US in 2018 for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years and older.
Presently, it is available in over 60 countries around the world.