Apellis Announces U.S. Food and Drug Administration (FDA) Approval of EMPAVELI (pegcetacoplan) for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)
EMPAVELI, the first targeted C3 therapy, is approved for use in adults with PNH who are:
- Treatment naïve
- Switching from C5 inhibitor Soliris (eculizumab)
- Switching from C5 inhibitor Ultomiris (ravulizumab)
EMPAVELI was superior to Soliris for the change from baseline in hemoglobin level at Week 16 in the Phase 3 PEGASUS study
85% of patients treated with EMPAVELI were transfusion free compared to 15% on Soliris at Week 16; EMPAVELI met non-inferiority compared to Soliris on transfusion avoidance
Apellis to host investor conference call on Monday, May 17 at 8:00 a.m. ET
WALTHAM, Mass., May 14, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI (pegcetacoplan), the first and only targeted C3 therapy for treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is approved for use in adults with PNH who are treatment naïve as well as patients switching from the C5 inhibitors Soliris (eculizumab) and Ultomiris (ravulizumab).
“EMPAVELI has the potential to improve the lives of patients with PNH by increasing hemoglobin and reducing blood transfusion requirements,” said Olga Frankfurt, M.D., PEGASUS study investigator and associate professor in the department of medicine, division of hematology and oncology, Robert H. Lurie Comprehensive Cancer Center of Northwestern University. “Through my work as an investigator on the PEGASUS study, I was excited to see EMPAVELI providing broad control of PNH."
“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,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. “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.”