Apellis Announces U.S. Food and Drug Administration (FDA) Approval of EMPAVELI (pegcetacoplan) for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) - Seite 2

The approval of EMPAVELI is based on results from the head-to-head Phase 3 PEGASUS study, which were recently published in the New England Journal of Medicine. In the PEGASUS study, EMPAVELI met the primary endpoint, demonstrating superiority to Soliris for the change from baseline in hemoglobin level at Week 16 with an adjusted mean increase of 3.84 g/dL of hemoglobin (p<0.0001). Additionally, EMPAVELI met non-inferiority compared to Soliris on the endpoint of transfusion avoidance. Eighty five percent of EMPAVELI-treated patients were transfusion free over 16 weeks versus 15% of Soliris-treated patients.

“We are pleased to hear of the FDA’s decision to approve EMPAVELI, which is an important milestone for patients,” said Janice Frey-Angel, chief executive officer and executive director, Aplastic Anemia and MDS International Foundation (AAMDSIF). “Many PNH patients are seeking choices in their treatment, so the approval brings new promise for the PNH community.”

The prescribing information for EMPAVELI contains a boxed warning. EMPAVELI may increase the risk of meningococcal and other serious infections caused by encapsulated bacteria that may become rapidly life threatening or fatal if not recognized and treated early. A Risk Evaluation and Mitigation Strategy (REMS) has been approved by the FDA for EMPAVELI. Prescribers must counsel patients about the risk of serious infection, provide patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria.

The most common serious adverse reaction in patients treated with EMPAVELI was infections (5%). The most common adverse reactions (≥10%) with EMPAVELI were injection site reactions (39%), infections (29%), diarrhea (22%), abdominal pain (20%), respiratory tract infection (15%), viral infection (12%), and fatigue (12%). No cases of meningitis and no deaths were reported in patients treated with EMPAVELI.

PNH is a rare, chronic, life-threatening blood disorder caused by an acquired mutation, which leads to uncontrolled complement activation and the destruction of red blood cells through intravascular and extravascular hemolysis. According to a retrospective and a cross-sectional study of patients treated with C5 inhibitors, at least 72% had persistently low hemoglobin^1,2 and at least 36% required one or more transfusions a year.¹