Epysqli, Soliris biosimilar, favored for approval for PNH in EU

Recommendation based on studies, including Phase 3 SB12 trial of 50 patients

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by Steve Bryson PhD |

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European regulators have recommended approving Epysqli, a Soliris (eculizumab) biosimilar, to treat children and adults with paroxysmal nocturnal hemoglobinuria (PNH).

The recommendation came from the Committee for Medicinal Products for Human Use (CHMP) and will serve as the basis for a final approval by the European Commission.

Epysqli is a biosimilar referencing Soliris, an intravenous (into-the-vein) antibody-based therapy approved in Europe for adults and children with PNH regardless of a history of blood transfusions. A biosimilar is a biological product that’s highly similar to an already approved biological medicine and developed to be equivalent regarding quality, safety, and effectiveness.

“We are pleased to see Epysqli becoming our first biosimilar in hematology to be recommended for approval in Europe,” Byoungin Jung, vice president and regulatory affairs team leader at Samsung Bioepis, the company marketing Epysqli, said in a company press release. “This marks another significant milestone for Samsung Bioepis and more importantly for people living with PNH.”

PNH is an acquired disease marked by hemolysis, or blood cell destruction, leading to symptoms like blood in the urine, fatigue, shortness of breath, and abdominal pain.

Blood cell destruction in PNH is triggered by the activation of the complement system, a group of more than 30 immune proteins that, under normal circumstances, work together to eliminate harmful microbes.

Eculizumab, the active ingredient in Soliris and Epysqli, works by binding to complement protein C5, preventing its cleavage, and ultimately blocking the complement system’s activation.

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Most PNH patients on Soliris still experience symptoms, study says

CHMP’s opinion was based on evidence gathered from analytical, nonclinical, and clinical studies. It also included findings from the now completed Phase 3 SB12 study (NCT04058158), which compared the safety, efficacy, and pharmacological properties of Epysqli to Soliris.

The study enrolled 50 patients, ages 18 and older, who’d yet to be treated with eculizumab, at 27 sites in India, South Korea, Malaysia, Taiwan, Thailand, Romania, Ukraine, and Mexico.

Eligible participants had PNH-related symptoms or required blood transfusions in the year before joining the study. They also had higher than normal levels of lactate dehydrogenase (LDH), a marker of cell and tissue damage often used to assess PNH disease activity.

Participants were randomly assigned to receive 600 mg of intravenous Epysqli or Soliris every week for the first four weeks, followed by 900 mg for the fifth week, and then 900 mg every two weeks as a maintenance regimen. After 26 weeks (about six months), participants were switched to the opposing regimen until week 50 (about a year).

LDH levels measured at weeks 26 and 50 showed Epysqli was clinically equivalent to Soliris, according to Samsung Bioepis.

“We will continue our work to transform the way biologic therapies are brought to patients and enhance the lives of patients, including those with rare diseases, through our pioneering and innovative use of science and technology,” Jung said.