Takhzyro Approved for Pediatric Hereditary Angioedema
The approval was supported extrapolation of efficacy data from the phase 3 HELP study, along with safety and pharmacodynamic data from the phase 3 SPRING study.
The approval was supported extrapolation of efficacy data from the phase 3 HELP study, along with safety and pharmacodynamic data from the phase 3 SPRING study.
Using CRISPR/Cas9 technology, the investigational therapy is designed to edit disease-causing genes inside the human body.
The prefilled syringe is provided as a 300mg/2mL (150mg/mL) ready-to-use solution for subcutaneous injection and does not require additional reconstitution or dilution.