Apellis Announces U.S. Food and Drug Administration (FDA) Approval of EMPAVELI™ (pegcetacoplan) for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)
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.”
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 hemoglobin1,2 and at least 36% required one or more transfusions a year.1
Apellis is committed to helping patients with treatment access and support. ApellisAssist™ is a program designed to provide comprehensive product support to EMPAVELI patients throughout their treatment journey. This program provides services and product resources including insurance support, education, training, as well as financial assistance for eligible patients. Patients and healthcare providers can contact 1-866-692-7527 for more information.
A Marketing Authorization Application for pegcetacoplan for the treatment of PNH is under review by the European Medicines Agency with the potential for a European Commission decision in the second half of 2021.
Conference Call and Webcast
About the PEGASUS Study
The study was conducted in collaboration with SFJ Pharmaceuticals, who supported the development of EMPAVELI in PNH. SFJ is a global drug development company, which provides a unique and highly customized co-development partnering model for the world’s top pharmaceutical and biotechnology companies.
About EMPAVELI™ (pegcetacoplan)
U.S. Important Safety Information for EMPAVELI
BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA
WARNINGS AND PRECAUTIONS
Serious Infections Caused by Encapsulated Bacteria
For patients without known history of vaccination, administer required vaccines at least 2 weeks prior to receiving the first dose of EMPAVELI. If immediate therapy with EMPAVELI is indicated, administer required vaccine as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis.
Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider discontinuation of EMPAVELI in patients who are undergoing treatment for serious infections.
Monitoring PNH Manifestations after Discontinuation of EMPAVELI
Interference with Laboratory Tests
USE IN SPECIFIC POPULATIONS
Females of Reproductive Potential
About the Apellis and Sobi Collaboration
Apellis Forward-Looking Statement
Back Together Happy Hour - Welcome Networking Event - Sponsored by Telefonica
Conference Sessions with Lunch