FDA approves Samsung Bioepis’ Epsyqli biosimilar for PNH and aHUS treatment

This approval marks Samsung Bioepis’ eighth biosimilar approved by the FDA and aims to expand treatment options for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

PNH is a rare, life-threatening blood disorder characterized by the destruction of red blood cells, while aHUS is a genetic condition that causes abnormal blood clots in small blood vessels, leading to kidney failure and other severe complications. The availability of biosimilars like Epsyqli offers a critical opportunity to improve the quality of life for patients suffering from these debilitating conditions.

It is approved for reducing hemolysis in PNH patients and inhibiting complement-mediated thrombotic microangiopathy in aHUS patients. However, it is not indicated for treating patients with Shiga toxin E. coli-related hemolytic uremic syndrome (STEC-HUS).

The FDA’s approval of Epsyqli is based on comprehensive evidence, including analytical, non-clinical, and clinical data, demonstrating that Epsyqli is highly similar to Soliris with no clinically meaningful differences in safety, purity, or potency. A phase 1 study in healthy volunteers confirmed pharmacokinetics equivalence, while a phase 3 study in PNH patients demonstrated clinical equivalence in efficacy and safety between Epsyqli and Soliris.

Christopher Hansung Ko, president and CEO of Samsung Bioepis, said: “The FDA approval of Epsyqli is a significant milestone for the PNH and aHUS communities. Biosimilars have the potential to reduce healthcare costs and improve access to treatments, fulfilling our mission of providing quality-assured biologic medicines to patients in need.”

Eculizumab, the active ingredient in Epsyqli, is a well-established treatment for PNH and aHUS, rare diseases affecting approximately 50,000 and 5,000 people in the US, respectively. Despite its effectiveness, the high cost of treatment has been a barrier for many patients. Biosimilars like Epsyqli can alleviate this financial burden, providing more affordable options for biologic therapies.

Outside of the US, Epsyqli has also been approved by the European Commission (EC) and Korea’s Ministry of Food and Drug Safety (MFDS) for treating PNH and aHUS. In regions where Epsyqli is available, it may not be prescribed for other indications for which Soliris is approved.

Epsyqli is a complement inhibitor used for the treatment of patients with PNH to reduce hemolysis and patients with aHUS to inhibit complement-mediated thrombotic microangiopathy. It is not indicated for treating patients with STEC-HUS. The monoclonal antibody and anti-C5 complement inhibitor eculizumab has become a standard treatment for these rare diseases, though its high cost has been a significant barrier to access.

With the FDA approval of Epsyqli, Samsung Bioepis aims to provide a more affordable and accessible treatment option for patients with PNH and aHUS. This development is expected to relieve some of the financial burden on the healthcare system and offer patients a quality-assured alternative to the reference biologic.