The US FDA approval of Empaveli is based on results from the head-to-head Phase 3 PEGASUS study
EMPAVELI single-dose carton and vial. (Credit: Apellis Pharmaceuticals)
Apellis Pharmaceuticals has received the US Food and Drug Administration (FDA) approval for its Empaveli (pegcetacoplan) to treat adults with paroxysmal nocturnal hemoglobinuria (PNH).
Empaveli is said to be the first targeted C3 therapy that is approved for use in adults with PNH who are treatment naïve, switching from C5 inhibitor Soliris (eculizumab) and switching from C5 inhibitor Ultomiris (ravulizumab).
PNH is a rare, chronic, life-threatening disease of the blood. It is caused by an acquired mutation that leads to uncontrolled complement activation and the destruction of red blood cells.
Apellis co-founder and CEO Cedric Francois said: “As the first, FDA-approved targeted C3 therapy, Empaveli has the potential to redefine treatment for adults with PNH, including patients switching from any C5 inhibitor and treatment-naïve patients.
“Thank you to the clinical trial participants, PNH community, investigators, healthcare professionals, SFJ Pharmaceuticals, and more who helped contribute to this significant milestone.
“This approval represents a major scientific advancement as EMPAVELI ushers in the first new class of complement medicine in almost 15 years. We look forward to exploring the full potential of targeting C3 and continue to advance registrational programs of this therapy across multiple complement-driven diseases with high unmet need.”
The US regulator’s approval of Empaveli is based on results from the head-to-head Phase 3 PEGASUS study. The study results were recently published in the New England Journal of Medicine.
The study revealed that Empaveli met the primary endpoint, presenting superior results compared to Soliris for the change from baseline in haemoglobin level at week 16.
Empaveli also met non-inferiority compared to Soliris on the endpoint of transfusion avoidance.
Apellis stated that 85% of the patients treated with Empaveli were transfusion free over 16 weeks compared to 15% of the patients treated with Soliris.
The company said that a marketing authorisation application for pegcetacoplan for the treatment of PNH is under review by the European Medicines Agency. The decision from the European Commission is expected in the second half of 2021.