The US FDA has accepted the sBLA for Priority Review of Evkeeza as an adjunct to other lipid-lowering therapies in children aged 5-11yrs. with HoFH. The US FDA’s decision is expected on March 30, 2023
The sBLA was based on the results from a three-part trial (parts A/B/C) evaluating Evkeeza. The trial met its 1EPs i.e., 48% reduction in LDL-C @24wk. by adding Evkeeza to other lipid-lowering therapies, patients treated with Evkeeza achieved 79% LDL-C reduction @24wk. with an absolute reduction in LDL-C from a baseline of 132mg/dL
The safety profile was consistent with that observed in adults & pediatric patients aged ≥12yrs. Evkeeza has been approved in the US, EU & the UK for patients aged ≥12yrs. with HoFH
Ref: Regeneron | Image: Regeneron
