FDA Approves On-Body Injector for Self Administration of Pegcetacoplan for PNH

The FDA has approved the Empaveli Injector designed to enhance the self administration of pegcetacoplan (Empaveli), which is approved for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).¹

At 20 mL, the compact, single-use, on-body injector (OBI) is the first high-volume, subcutaneous, on-body delivery system for pegcetacoplan. The OBI features a push button to start the injection, and a hidden needle automatically retracts after the dose is given. The Empaveli Injector was developed in collaboration with Enable Injections, based on the enFuse® Syringe Transfer System.

“The approval of the Empaveli Injector is an exciting advancement. People living with PNH can now receive the benefits of [pegcetacoplan] via on-body treatment administration, allowing for greater mobility,” Carlos de Castro, MD, professor of medicine at Duke University, stated in a news release. “With the Empaveli Injector, patients can seamlessly integrate [pegcetacoplan] treatment into their daily lives, whether that is at home or on the go.”

In May 2021, the FDA approved pegcetacoplan for the treatment of adults with PNH who are treatment naïve and those who are switching from the C5 inhibitors eculizumab (Soliris) and ravulizumab (Ultomiris).²

The approval was supported by data from the phase 3 PEGASUS trial (NCT03500549), in which pegcetacoplan demonstrated superiority to eculizumab regarding the change in hemoglobin level from baseline to week 16, with an adjusted mean increase in hemoglobin of 3.84 g/dL (95% CI, 2.33-5.34; P < .0001), meeting the trial’s primary end point.³

Additionally, 85% of patients treated with pegcetacoplan (n = 35/41) no longer required transfusions, compared with 15% of patients given eculizumab (n = 6/39).

The most common adverse effects reported during the study included injection-site reactions (37% vs 3% for pegcetacoplan and eculizumab, respectively), diarrhea (22% vs 3%), breakthrough hemolysis (10% vs 23%), headache (7% vs 23%), and fatigue (5% vs 15%).

The open-label, phase 3 PEGASUS trial enrolled patients at least 18 years of age with PNH who had hemoglobin levels lower than 10.5 g/dL despite at least 3 months of treatment with eculizumab.

After all patients received treatment with subcutaneous pegcetacoplan at 1080 mg twice per week plus eculizumab during a 4-week run-in period, they were randomly assigned 1:1 to receive pegcetacoplan monotherapy or eculizumab monotherapy followed by pegcetacoplan plus eculizumab during a 16-week controlled period. All patients then received single-agent pegcetacoplan during a 32-week open-label period.

Along with the primary end point of change in hemoglobin level from baseline to week 16, key secondary end points included the proportion of patients who did not require a transfusion during the randomized, controlled period, as well as the change from baseline to week 16 in absolute reticulocyte count, lactate dehydrogenase level, and Functional Assessment of Chronic Illness Therapy–Fatigue score.

“[Pegcetacoplan] continues to demonstrate its potential to elevate the standard of care, including rapid and sustained improvements of PNH disease measures. Now, we are further enhancing the patient experience with the approval of the Empaveli Injector, an innovative and first-of-its-kind, high-volume injector,” Peter Hillmen, MB ChB, PhD, head, rare disease advisor at Apellis Pharmaceuticals, stated in a news release.1“This approval is an example of how we are continually innovating with patients at the forefront.”

References

1. Apellis announces US FDA approval of the Empaveli Injector, a device to streamline self-administration. News release. Apellis Pharmaceuticals. October 2, 2023. Accessed October 2, 2023. https://investors.apellis.com/news-releases/news-release-details/apellis-announces-us-fda-approval-empavelir-injector-device
2. FDA approves new treatment for adults with serious rare blood disease. News release. FDA. May 18, 2021. Accessed October 2, 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-treatment-adults-serious-rare-blood-disease
3. Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384(11):1028-1037. doi:10.1056/NEJMoa2029073