The approval was based on data from a phase 3 study that evaluated Trikafta in 66 children 6 to 11 years of age with CF who were either homozygous for the F508del mutation or heterozygous for the F508del mutation and 1 minimal function mutation.
The approval was based on data from a pivotal phase 2/3 trial that assessed Ryplazim in 15 adult and pediatric patients with hypoplasminogenemia.
BBP-631 is an adeno-associated virus 5 (AAV5) gene therapy designed to deliver a functional copy of the 21-hydroxylase gene to restore the body’s ability to produce cortisol and aldosterone.
Losmapimod is a selective p38α/β mitogen activated protein kinase inhibitor that works by reducing the expression of the DUX4 gene in muscle cells.
