FDA approves Epysqli, biosimilar of Soliris, to treat PNH
Treatment joins Bkemv, another biosimilar, as an option for patients in the US
The U.S. Food and Drug Administration (FDA) has approved Epysqli (eculizumab-aagh), a Soliris (eculizumab) biosimilar, to prevent red blood cell destruction (hemolysis) in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Biosimilars contain a version of the active ingredient of an approved biological therapy — a treatment that uses substances from living organisms — and have a nearly identical safety and efficacy profile to the reference product. These medications usually come at a lower price than the original reference therapy, but they carry the same clinical benefits.
The FDA’s decision to approve Epysqli for PNH was supported by preclinical and clinical evidence submitted by its developer, Samsung Bioepis, showing no clinically meaningful differences between its therapy and Soliris in terms of safety, purity, or potency.
As a biosimilar, Epysqli is expected to cost less than Soliris
Epysqli was approved as a Soliris biosimilar in the European Union and Korea. In all three regions, regulatory decisions also covered Epysqli’s use in atypical hemolytic uremic syndrome (aHUS), another condition that Soliris is approved to treat.
“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, president and CEO at Samsung Bioepis, said in a company press release.
In PNH, the immune system’s complement cascade drives the destruction of healthy blood cells.
Eculizumab, the active ingredient in Soliris and Epysqli, works by binding to an important complement system protein called C5 in order to block complement activation. This helps to reduce complement-mediated hemolysis and related PNH symptoms in patients.
Soliris, marketed by AstraZeneca, was approved to treat adults with PNH in 2007. It is also approved for other diseases where the complement system is implicated, including aHUS, neuromyelitis optica spectrum disorder, and myasthenia gravis.
Amgen’s Bkemv (eculizumab-aeeb) was approved by the FDA as the first interchangeable biosimilar to Soliris for treating PNH in May. It also is available in Europe under the brand name Bekemv.
Clinical trials established that Epysqli did not meaningfully diverge in safety and efficacy from Soliris, supporting its biosimilar status.
Epysqli showed similar safety and efficacy to Soliris in clinical trials
A randomized Phase 1 clinical trial (NCT03722329) in healthy volunteers showed that Epysqli and Soliris had similar pharmacological profiles and comparable safety and tolerability, according to the company.
Then, a Phase 3 study (NCT04058158) directly compared the effects of Epysqli against Soliris in adults with PNH new to treatment with a complement inhibitor. Fifty participants were randomly assigned to either of the two medications for about six months, after which the groups switched — those on Soliris moved to Epysqli and vice versa — for another six months.
In both groups, treatment was given via intravenous (into-the-vein) infusions once a week at a 600 mg dose for the first four weeks, 900 mg in the fifth week, followed by 900 mg every two weeks thereafter. This is the approved PNH dosing regimen for both therapies.
Trial findings showed that Epysqli was clinically equivalent to Soliris in terms of safety, efficacy, and pharmacological properties.
Levels of lactate dehydrogenase, a marker of cell and tissue damage reflective of disease activity in PNH, were similar with both treatments, meeting the study’s main goal. Moreover, similar proportions of patients on either treatment — a little over two-thirds — did not need blood transfusions.
“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,” Ko said.
Because the two medications are designed to be functionally equivalent, Epysqli has the same general risk profile as Soliris. Its prescribing label comes with a boxed warning for serious, life-threatening meningococcal infections, which have been reported in people treated with complement inhibitors. It also is available only through a restricted access program, called Epysqli REMS.
Similar to Soliris, Epysqli is contraindicated, or not recommended, for people with unresolved infections caused by the Neisseria meningitidis meningococcal bacteria.
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