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FDA approves Apellis’ Empaveli for rare blood disease

PEGASUS study results show Empaveli is better than Soliris at improving haemoglobin levels

The US Food and Drug Administration (FDA) has approved Apellis’ Empaveli for the treatment of adults with the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH), setting up a rivalry with Alexion’s Soliris.

The approval is based on the head-to-head PEGASUS study, which showed that complement C3 inhibitor Empaveli (pegcetacoplan) was better than Soliris (eculizumab) at improving haemoglobin levels in adults with PNH, an autoimmune disease characterised by the destruction of red blood cells and anaemia.

This study enrolled 80 adult PNH patients who had been on intravenous Soliris and were stable for at least three months with a haemoglobin level of less than 10.5 g/dL at baseline.

Patients received twice-weekly subcutaneous doses of Empaveli in addition to Soliris over four weeks and were then randomised to receive Apellis’ drug or their current Soliris dose for the main treatment phase of the trial.

After 16 weeks, haemoglobin levels in the Empaveli treatment group had increased by 3.8 g/dL compared to the Soliris group, equating to a 2.4 g/dL rise for the drug as opposed to a 1.5 g/dL decline with Alexion’s drug.

In addition, 85% of Empaveli-treated patients were transfusion free over 16 weeks compared with 15% of Soliris-treated patients.

“As the first, FDA-approved targeted C3 therapy, Empaveli has the potential to redefine treatment for adults with PNH, including patients switching from any C5 inhibitor and treatment-naïve patients. Thank you to the clinical trial participants, PNH community, investigators, healthcare professionals, SFJ Pharmaceuticals and more who helped contribute to this significant milestone,” said Cedric Francois, co-founder and chief executive officer, Apellis.

“This approval represents a major scientific advancement as Empaveli ushers in the first new class of complement medicine in almost 15 years. We look forward to exploring the full potential of targeting C3 and continue to advance registrational programmes of this therapy across multiple complement-driven diseases with high unmet need,” he added.

Whereas Alexion’s Soliris and its follow-up therapy Ultomiris target the C5 pathway, Apellis’ drug targets the compliment protein C3 – the central protein in the complement cascade.

Apellis is also conducting a study of Empaveli in treatment-naïve PNH patients – meaning they have not previously received a complement inhibitor within three months before entering the trial.

Article by
Lucy Parsons

17th May 2021

From: Regulatory



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