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EMPAVELI® (pegcetacoplan) Provided Long-Term Control of PNH in New Data Presented at ASH Annual Meeting

  • Rapid improvements in key markers of disease were sustained for up to three years across all adults with PNH
  • Majority of patients remained transfusion free over the long term
  • Zero cases of meningococcal infection observed over three years in this analysis
  • Data shared during an oral presentation

WALTHAM, Mass., Dec. 11, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi today presented post hoc data that reinforce the long-term efficacy and safety of EMPAVELI® (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) for up to three years. The data were reported during an oral presentation at the American Society of Hematology (ASH) Annual Meeting.

“These results show that treatment with EMPAVELI can help PNH patients achieve rapid and sustained control of their disease over the long term,” said Carlos de Castro, M.D., presenting author and professor of medicine, Duke University. “Furthermore, it is impressive that the majority of patients remained transfusion free up to three years, alleviating a significant and common disease burden for many people living with PNH.”

The analysis integrated data across the Phase 3 PEGASUS and PRINCE studies and the long-term extension study. After starting treatment with EMPAVELI, key markers of disease rapidly improved and were sustained in both treatment-naïve patients and patients previously treated with eculizumab. Improvements in hemoglobin reached normal or near-normal levels, and mean lactate dehydrogenase (LDH) was maintained below the upper limit of normal.

Additionally, 67% of treatment-naïve patients from PRINCE were transfusion free for up to 2.5 years, and 52% of patients from PEGASUS remained transfusion free for up to 3 years. Less than 25% of patients were transfusion free in the year prior to entering the PRINCE and PEGASUS studies.

The safety profile was consistent with previous clinical study results, and no new or unexpected safety findings were identified. Approximately 4.5% of patients experienced a serious adverse event deemed related to treatment with pegcetacoplan. No meningococcal infections were reported.

The prescribing information for EMPAVELI contains a boxed warning, which states that 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.

“The strength of these long-term EMPAVELI results highlight why we are seeing compliance rates of 97% with real-world use,” said Peter Hillmen, M.B. Ch.B., Ph.D., head, rare disease advisor, Apellis. “By achieving and maintaining normal clinical measures over time, EMPAVELI has the potential to elevate the standard of care for all adults with PNH."

About the Long-Term Efficacy and Safety Extension (APL2-307) Study
The APL2-307 study was a nonrandomized, multicenter, open-label Phase 3 extension study of 137 adults with paroxysmal nocturnal hemoglobinuria (PNH) who completed previous EMPAVELI®/Aspaveli® (pegcetacoplan) Phase 1 (PHAROAH, PADDOCK), Phase 2 (PALOMINO), and Phase 3 (PEGASUS, PRINCE) clinical trials. Patients in these studies were either anemic despite eculizumab treatment or were naïve to complement inhibitors. During the trial, patients continued to receive 1080 mg of EMPAVELI twice weekly or once every three days (PEGASUS, PRINCE) or switched to 1080 mg of EMPAVELI twice weekly (PHAROAH, PADDOCK, PALOMINO). The primary objective was to establish the long-term efficacy and safety of EMPAVELI.

About the PEGASUS Study
The PEGASUS study (APL2-302; NCT03500549) was a randomized, multi-center, head-to-head Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI®/Aspaveli® (pegcetacoplan) compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of EMPAVELI twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of EMPAVELI twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label EMPAVELI treatment period.

About the PRINCE Study
The PRINCE study (NCT04085601) was a randomized, multi-center, open-label, controlled Phase 3 study in 53 treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI®/Aspaveli® (pegcetacoplan) in patients who had not received treatment with any complement inhibitor within three months prior to screening. During the 26-week randomized, controlled period, patients received either 1080 mg of EMPAVELI twice weekly or standard of care therapy, which did not include complement inhibitors. Patients in the standard of care group had the option to escape to the EMPAVELI group if their hemoglobin decreased by 2 g/dL or more from their baseline value.

About EMPAVELI®/Aspaveli® (pegcetacoplan)
EMPAVELI®/Aspaveli® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for several other rare diseases across hematology and nephrology.

U.S. Important Safety Information for EMPAVELI


  • Meningococcal infections may occur in patients treated with EMPAVELI and may become rapidly life-threatening or fatal if not recognized and treated early. Use of EMPAVELI may predispose individuals to serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B.
  • Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria.
  • Vaccinate patients at least 2 weeks prior to administering the first dose of EMPAVELI unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection.
  • Vaccination reduces, but does not eliminate, the risk of serious infections. Monitor patients for early signs of serious infections and evaluate immediately if infection is suspected.
  • EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the EMPAVELI REMS, prescribers must enroll in the program.


  • Hypersensitivity to pegcetacoplan or to any of the excipients
  • Not currently vaccinated against certain encapsulated bacteria, unless the risks of delaying EMPAVELI treatment outweigh the risks of developing a bacterial infection with an encapsulated organism
  • Unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae


Serious Infections Caused by Encapsulated Bacteria
The use of EMPAVELI may predispose individuals to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib). To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI.

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.

Because of the risk of serious infections, EMPAVELI is available only through a restricted program under a REMS. Under the EMPAVELI REMS, prescribers must enroll in the program and must counsel patients about the risk of serious infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria. Enrollment and additional information are available by telephone: 1-888-343-7073 or at

Infusion-Related Reactions
Systemic hypersensitivity reactions (e.g., facial swelling, rash, urticaria) have occurred in patients treated with EMPAVELI. One patient (less than 1% in clinical studies) experienced a serious allergic reaction which resolved after treatment with antihistamines. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved.

Monitoring PNH Manifestations after Discontinuation of EMPAVELI
After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues EMPAVELI for at least 8 weeks to detect hemolysis and other reactions. If hemolysis, including elevated LDH, occurs after discontinuation of EMPAVELI, consider restarting treatment with EMPAVELI.

Interference with Laboratory Tests
There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels.

Most common adverse reactions in patients with PNH (incidence ≥10%) were injection-site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, pain in extremity, hypokalemia, fatigue, viral infection, cough, arthralgia, dizziness, headache, and rash.


Females of Reproductive Potential
EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose.

Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria, and difficulty breathing (dyspnea).

About the Apellis and Sobi Collaboration
Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.

About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first and only therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across serious retinal, rare, and neurological diseases. For more information, please visit or follow us on Twitter and LinkedIn.

Apellis Forward-Looking Statement
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “can,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 21, 2023 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Media Contact: 
Lissa Pavluk

Investor Contact: 
Meredith Kaya

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