FDA approves Epysqli, biosimilar of Soliris, as aHUS treatment
Decision based on data including evidence from PNH trial
The U.S. Food and Drug Administration (FDA) has approved Epysqli (eculizumab-aagh), a biosimilar to Soliris (eculizumab), as an atypical hemolytic uremic syndrome (aHUS) treatment.
Epysqli, from Samsung Bioepis, has also been approved to treat people with paroxysmal nocturnal hemoglobinuria (PNH), a rare disease that, like aHUS, occurs when the complement system that forms part of the body’s immune defenses becomes overly active, causing red blood cells to break down (hemolysis). The medication is not indicated for people with typical HUS, also called STEC-HUS, a disorder caused by toxins produced by certain bacteria, most commonly Escherichia coli.
Epysqli joins other Soliris biosimilars previously approved. Bkemv (eculizumab-aeeb) this year became the first interchangeable biosimilar of Soliris to become available in the U.S. for aHUS and PNH. A biosimilar is a medication that’s similar to an existing, approved biological medicine, which is called the reference medicine. It’s designed to have the same properties as the reference medicine, and to have similar safety and effectiveness. But like a generic medicine, it’s often cheaper than the original medication.
“The FDA approval of Epysqli as a biosimilar to Soliris marks an important milestone for PNH and aHUS communities since biosimilars have a potential to positively impact patients and healthcare systems by reducing healthcare costs and improving access to treatments,” Christopher Hansung Ko, PhD, president and CEO at Samsung Bioepis, said in a company press release.
Epysqli was previously approved in Europe and South Korea.
Antibody therapy as aHUS treatment
“Our mission has been, and always will be improving the lives of patients by providing quality-assured, safe and effective biologic medicines, and our work to fulfill this mission is expanding into rare disease areas where patients continue to suffer from limited access to life-enhancing medicines,” Hansung Ko said.
Originally developed by Alexion Pharmaceuticals, later acquired by AstraZeneca, Soliris is an antibody therapy. Eculizumab, the active ingredient, targets a complement protein called C5. By binding to C5, eculizumab is designed to prevent formation of the C5b-9 complement protein complex and stop clotting within small blood vessels.
The FDA’s decision was based on evidence from non-clinical and clinical data, including a Phase 1 trial (NCT03722329) in healthy volunteers and a Phase 3 trial (NCT04058158) in PNH patients, which supported similar efficacy and safety to Soliris.
The Phase 1 trial showed that Epysqli was equivalent to Soliris with regard to the therapy’s safety, pharmacokinetics, and immune profile. Pharmacokinetics refers to the movement of a therapy into, through, and out of the body.
In the Phase 3 study, patients with PNH were randomly assigned to receive the same dose of Epysqli or Soliris for 26 weeks (about six months), after which they switched treatments over a similar period. Results showed Epysqli to be therapeutically equivalent to Soliris in its ability to reduce hemolysis and the need for blood transfusions.
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