New study backs Empaveli as a safe, effective treatment for PNH
Injection therapy found to help patients with red blood cell destruction
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Use of the approved injection therapy Empaveli (pegcetacoplan) appears to be effective and generally safe for people with paroxysmal nocturnal hemoglobinuria (PNH) — especially for patients who, despite treatments, have ongoing destruction of red blood cells, known as hemolysis.
Those are the findings of a new analysis from researchers in Brazil, who reviewed five studies that tracked the therapy’s use in both clinical trials and the real world.
According to the team, Empaveli appeared to be particularly effective for PNH patients with ongoing hemolysis despite use of a C5 inhibitor, a targeted medication that blocks activation of a protein in the body’s immune system.
Empaveli “demonstrates consistent efficacy signals across key [blood-related] endpoints and an acceptable safety profile, supporting its potential role as an important therapeutic option,” the researchers wrote, noting the therapy’s effectiveness in “uniquely targeting” one type of hemolysis “while maintaining protection against” another.
“Further studies, including those in implementation science and health economics, are required to determine its optimal deployment across diverse care ecosystems,” the researchers wrote.
The study, “Efficacy and Safety of the C3 Inhibitor Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Meta-Analysis,” was published in the European Journal of Haematology.
PNH is mainly caused by genetic mutations that make red blood cells vulnerable to attack by the body’s complement system, a part of the immune system. As a result, red blood cells are destroyed too early, leading to symptoms such as blood in the urine, blood clots, and fatigue caused by anemia, or too few red blood cells in the body.
Empaveli, developed by Apellis and Sobi, works by inhibiting C3, a protein in the complement system. Unlike other treatments that inhibit the complement system at a later stage, Empaveli helps protect red blood cells from hemolysis both inside and outside blood vessels, increasing hemoglobin — the protein that carries oxygen — and reducing the need for blood transfusions.
Researchers note Empaveli can be used at home
Now, a quintet of researchers sought to assess the effectiveness and safety of the PNH treatment, which has been approved in the U.S. since 2021.
To collect evidence, the team searched four databases for studies in PNH. Five studies — two randomized, one single-arm, and two involving people with PNH in the real world — were ultimately used, covering a total of 271 patients. These individuals, followed for 16 to 32 weeks, or about 4-8 months, came from several international centers.
Some patients had never received treatment with a complement inhibitor. Others, meanwhile, had been previously treated with Soliris (eculizumab), a complement inhibitor that inhibits the C5 protein and acts at a later stage of the complement cascade than Empaveli.
Many of these individuals had long-standing disease, and some had aplastic anemia — occurring when the bone marrow fails to produce enough new red blood cells — or a history of frequent blood transfusions. In one study, nearly three-quarters of patients had received at least four blood transfusions in the previous year.
In most studies, patients received Empaveli as a 1,080 mg subcutaneous, or under-the-skin, injection twice weekly. In a few real-world cases, the dosing schedule was adjusted, while an earlier study tested lower doses of 270 to 360 mg daily.
During treatment, most patients (81%) did not require blood transfusions.
In general, across the studies, patients’ hemoglobin levels increased and remained between 11.5 and 12.8 g/dL. About six months after treatment, almost half of the patients (43%) reached normal hemoglobin counts, while two-thirds had normal counts of immature red blood cells known as reticulocytes. Normal levels of lactate dehydrogenase, a marker of hemolysis, were achieved for 69% of patients, the data showed.
Side effects common, but PNH treatment was generally safe
Side effects were reported for most patients (84%). The most common were fever, headache, and dizziness. Other reported side effects included low potassium and joint pain. Serious side effects occurred in a smaller proportion of patients (17%). There were 19 reports of hemolysis, including breakthrough hemolysis, which can occur suddenly despite treatment with a complement inhibitor.
According to the researchers, “this systematic review synthesized the evidence regarding the efficacy and safety of [Empaveli] in treating PNH.” Overall, the team found the therapy met those goals, also noting its “comprehensive hemolysis suppression.”
The researchers also noted the convenience of Empaveli use at home, noting “its subcutaneous self-administration paradigm fundamentally shifts treatment delivery from hospital dependency to patient empowerment.”
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