EPYSQLI® (Eculizumab-aagh) — A Landmark Biosimilar in Rare Disease Treatment

Introduction

Samsung Bioepis’ EPYSQLI® (eculizumab-aagh) has earned FDA approval as a biosimilar to Soliris® (eculizumab). Approved on July 19, 2024, EPYSQLI offers a more affordable alternative for treating debilitating rare diseases.FDA Access DataSamsung BioepisU.S. Pharmacist

Indications

EPYSQLI is indicated for:

• Paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.
• Atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.FDA Access DataSamsung BioepisU.S. Pharmacist
• In November 2024, its approval expanded to include generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor antibody (AchR) positive.tevapharm.comU.S. Pharmacist

Efficacy & Clinical Performance

EPYSQLI matches the reference biologic in both efficacy and safety:

• A Phase I randomized, double-blind trial in healthy volunteers demonstrated equivalent pharmacokinetics (PK), pharmacodynamics (PD), safety, and immunogenicity to Soliris.Samsung Bioepis
• A Phase III, double-blind, multicenter crossover trial in PNH patients confirmed clinical equivalence in efficacy, safety, PK, and immunogenicity.Samsung Bioepis
• Post-hoc subgroup analyses in PNH showed comparable outcomes in transfusion avoidance and hemoglobin stabilization across both Asian and non-Asian groups, validating global applicability.PMC
• For aHUS, biosimilar efficacy is supported by data indicating improvements in thrombotic microangiopathy and renal function, including dialysis independence in some patients.medicalpolicy.bcbstx.comWikipedia

Cost & Access Advantage

Through a strategic partnership, Teva and Samsung Bioepis launched EPYSQLI in the U.S. at a 30% discount compared to Soliris wholesale acquisition cost (WAC).tevapharm.comU.S. Pharmacist This pricing helps enhance accessibility for patients with rare diseases, reducing financial barriers.

Patent & Regulatory Landscape

As a biosimilar, EPYSQLI stands on the foundation of Soliris’ patent legacy, navigating through exclusivity for market entry. Its biosimilarity has cleared the way for adoption upon Soliris patent expiry.U.S. PharmacistWikipedia

EPYSQLI is in the pipeline to become an interchangeable biosimilar in the U.S., further expanding users’ flexibility once interchangeability status is granted.tevapharm.comU.S. Pharmacist

Safety Considerations

Like Soliris, EPYSQLI requires risk mitigation due to its mechanism of complement inhibition:

• Increased risk of meningococcal infections—patients must be vaccinated according to ACIP guidelines prior to treatment initiation.
• EPYSQLI is available via a REMS (Risk Evaluation and Mitigation Strategy) program to ensure safe use.Samsung BioepisAetna

Conclusion

EPYSQLI represents a significant advancement in biologic therapy for rare, life-threatening diseases. By delivering comparable efficacy and safety to Soliris while improving cost-accessibility, it marks a notable milestone in biosimilar innovation.