FDA clears injector aiming to make Empaveli use more convenient

Single-use device allows PNH patients to move about during dosing

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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The U.S. Food and Drug Administration (FDA) has cleared for use an injector device intended to make self-administration of Empaveli (pegcetacoplan) more convenient for adults with paroxysmal nocturnal hemoglobinuria (PNH).

Empaveli, marketed as Aspaveli in Europe, is given via under-the-skin (subcutaneous) injections. Before the FDA decision, patients were able to self-infuse the medication using a commercially available infusion pump.

The single-use device, called the Empaveli Injector, is designed to make the dosing process more convenient. With an adhesive backing, it’s placed directly on the abdomen and the medication is released by pushing a button on the device.

Patients can then continue with activities while the medicine is injected, a process that takes about 30 to 60 minutes. Its hidden needle will retract once dosing is complete, and the device can be removed and properly discarded.

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No change in dosing regimen when Empaveli given with injector device

“People living with PNH can now receive the benefits of Empaveli via on-body treatment administration, allowing for greater mobility,” Carlos de Castro, MD, professor of medicine at Duke University in North Carolina, said in a press release.

“With the Empaveli Injector, patients can seamlessly integrate Empaveli treatment into their daily lives, whether that is at home or on the go,” added de Castro, a study investigator for the Phase 3 PEGASUS trial (NCT03500549) that supported Empaveli’s regulatory approvals.

Empaveli was co-developed by Apellis Pharmaceuticals and Sobi, and its injector device was developed in collaboration with Enable Injections.

The injector uses the same single-dose vials of Empaveli that are commercially available, and it requires no change in the therapy’s dosing regimen. The treatment is given at a recommended dose of 1,080 mg in 20 mL of fluid twice weekly.

Once the injector is filled with the medication, it is placed on the stomach, about an inch away from the belly button and from any previous injection site.

After the start button is pushed, the injection will take between a half-hour and an hour to complete. This is the same amount of time as the previous administration method using an external infusion pump, but it is expected to be more convenient, allowing people to move about while being treated.

The injector device is intended for one-time use, meaning that a new one will be used for each dose administered.

Patients need to be shown how to fill and use the Empaveli Injector by a healthcare provider prior to self-administering the medication, and they should never attempt to use the Empaveli Injector or the commercial infusion pump prior to being instructed in its proper use.

It’s normal for the skin to be red after removing the device from the abdomen, Apellis notes on the page detailing its use. If redness does not go away after a couple days, patients inform their doctor. It is not normal for the skin to be bumpy or irritated during an injection, as this may indicate a sensitivity to the adhesive backing or the medication. A doctor should be notified right away in these cases.

Empaveli works by inhibiting C3, a protein that’s part of the immune system’s complement pathway. This pathway is thought to underlie the immune attack that drives red blood cell destruction in PNH. By inhibiting C3 and suppressing complement activation, Empaveli is expected to help ease patients’ anemia — a lack of healthy red blood cells — and associated PNH symptoms.

The therapy was approved by the FDA in 2021 to treat adults with PNH. It is also approved in Europe for adults with PNH who are anemic despite treatment with a C5 complement inhibitor therapy, like Soliris or Ultomiris, for at least three months.

“Empaveli continues to demonstrate its potential to elevate the standard of care, including rapid and sustained improvements of PNH disease measures,” said Peter Hillmen, PhD, lead rare disease advisor at Apellis. “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.”