Apellis Gains Momentum in Battle to Unseat Alexion in PNH with FDA Approval


Shares of Apellis Pharmaceuticals fell more than 3% Friday, but rallied and gained more than $5 per share in after-market trading following the company’s announcement the U.S. Food and Drug Administration (FDA) approved Empaveli for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Empaveli (pegcetacoplan) is the first and only targeted C3 therapy for the treatment of adults with PNH, the Waltham, Mass.-based company said in its announcement. Empaveli is approved for use in PNH patients who are treatment naïve as well as patients switching from the C5 inhibitors Soliris (eculizumab) and Ultomiris (ravulizumab). 

On a PNH dedicated website maintained by Apellis, the company said up to 89% of patients treated with C5 inhibition showed varying signs of ongoing hemolysis. 

The PNH treatment was approved based on data from the Phase III PEGASUS study that showed pegcetacoplan exhibited over Soliris in improving hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH). New data from the focal study showed that pegcetacoplan’s effect was seen consistently over the study population, both in patients with low or no transfusion requirements and high transfusion requirements. 

Source: BioSpace

Pegcetacoplan also displayed a robust response across several key hematologic and clinical measures for PNH. The study showed that 71% of pegcetacoplan-treated patients attained LDH normalization compared to 15% of eculizumab-treated patients. Additionally, 73% of pegcetacoplan-treated patients achieved a clinically meaningful improvement in FACIT-fatigue score compared to 0% of eculizumab-treated patients, the company said. Full data from the study were published in the New England Journal of Medicine

Olga Frankfurt, lead PEGASUS investigator and associate professor in the department of medicine, division of hematology and oncology, Robert H. Lurie Comprehensive Cancer Center of Northwestern University, said Empaveli has the potential to improve the lives of PNH patients by increasing hemoglobin and reducing blood transfusion requirements. 

PNH is a rare blood disease where the red blood cells are destroyed by a part of the body’s immune system known as the complement system. According to a retrospective and a cross-sectional study of patients treated with C5 inhibitors, at least 72% had persistently low hemoglobin1,2, and at least 36% required one or more transfusions a year, Apellis said.

Cedric Francois, co-founder and chief executive officer of Apellis, said as the first-approved targeted C3 therapy for PNH, Empaveli has the potential to redefine treatment for patients battling this hematological disease. 

This approval represents a significant scientific advancement as Empaveli ushers in the first new class of complementary 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,” Francois said in a statement. 

Empaveli does come with a boxed warning of potential risk of meningococcal and other serious infections caused by encapsulated bacteria. The FDA has approved a Risk Evaluation and Mitigation Strategy (REMS) for Empaveli, Apellis said. About 5% of patients treated with Empaveli during studies developed an infection, but no cases of meningitis were reported, the company added.

Apellis is seeking approval in Europe as well. A Marketing Authorization Application is under review by the European Medicines Agency. A decision is expected in the second half of 2021. 


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