Pegcetacoplan (PEG) sustains robust improvements in hemoglobin and lactate dehydrogenase levels, as well as fatigue in patients with paroxysmal nocturnal hemoglobinuria (PNH), according to results presented at the 64th ASH Annual Meeting and Exposition. PEG also reduces the need for transfusions in these patients.

PEG is the first approved C3 inhibitor for the treatment of adults with PNH. The efficacy of PEG in improving hemoglobin levels and other disease parameters in PNH has been shown in several clinical trials.

At the ASH meeting, a team of researchers led by Jens Panse, MD, from Aachen, Germany, presented 48-week data from their ongoing study on the long-term safety and efficacy of PEG for treating PNH.

The open-label, nonrandomized, multicenter phase 3 study enrolled 137 patients with PNH, aged 18 years and older who completed previous phase 1, 2, and 3 PEG trials. Of these 137 patients, 107 had received 48 weeks of treatment at the time of data cutoff on top of the treatment time in the parent study. Patients were exposed to 1080 mg of PEG twice a week for 74 to 96 weeks, depending on the clinical trial in which they were taking part. 

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The results showed that baseline hemoglobin and lactate dehydrogenase levels as well as Functional Assessment of Chronic Illness Therapy-fatigue scores were maintained throughout the study. Most patients (83.2%) achieved transfusion avoidance through week 48.

Most patients (73.7%) had an adverse event through week 48, 16.1% of which were related to PEG treatment; most consisted of hemolysis. Serious adverse events also consisted of hemolysis and were seen in 19.7% of patients, although none were related to PEG treatment and were all resolved. Injection site reactions were mostly mild and transient and were seen in 10.9% of patients. There were no thrombotic events or meningitis infections. One patient died due to a sudden cardiac event but this was not related to PEG treatment.

“Normalization of hematologic parameters is achievable with PEG,” the researchers concluded.


Patriquin CJ, Bogdanovic A, Griffin M, et al. Long-term safety and efficacy of pegcetacoplan treatment in adults with paroxysmal nocturnal hemoglobinuria. 64th ASH Annual Meeting and Exposition. New Orleans, LA, December 10-13, 2022. Poster number 1248.

Pegcetacoplan long term safety and efficacy extension study. Last updated July 26, 2021. Accessed November 21, 2022.