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Apellis’ and Sobi’s Treatment for PNH Approved in EU

December 17, 2021

The European Commission approved Apellis Pharmaceuticals’ and Swedish Orphan Biovitrum’s AB Aspaveli, the first and only targeted C3 therapy, for the treatment of adults with paroxysmal nocturnal hemoglobinuria who are anemic after treatment with a C5 inhibitor for at least three months.

Photo: Guido Oelkers, CEO and president at Sobi

PNH is a rare, chronic, and life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells through intravascular hemolysis and extravascular hemolysis. Characterized by persistently low hemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue caused by anemia. Despite improvements in hemolytic activity with C5 inhibitor treatment, approximately 72 percent of people with PNH treated with C5 inhibitors remain anemic, according to a retrospective and a cross-sectional study.

“The European Commission’s approval of Aspaveli is a milestone for people living with PNH across Europe,” said Guido Oelkers, CEO and president at Sobi. “The symptoms of PNH can significantly impact quality of life. In addition, despite current therapy, many people still require frequent blood transfusions. We are now working with EU member states to provide access to this important medicine as quickly as possible.”

Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Aspaveli is approved in the EU as an orphan drug for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are anemic after treatment with a C5 inhibitor for at least three months and in the United States as Empaveli for the treatment of adults with PNH. The therapy is also under investigation for several other rare diseases across hematology, nephrology, and neurology.

The approval is based on the results from the head-to-head PEGASUS phase 3 study, which evaluated the efficacy and safety of Aspaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anemia despite treatment with eculizumab. The full safety and efficacy results were published in The New England Journal of Medicine in March 2021.

“As the first and only targeted C3 therapy in Europe, Aspaveli has the potential to elevate the standard of care for patients living with PNH,” said Federico Grossi, chief medical officer of Apellis. “Today’s approval represents the first new class of complement medicines in Europe in over a decade, building on the launch of this important treatment in the United States.”

Based on the recommendation from the European Medicines Agency’s Committee for Orphan Medicinal Products, the EC determined that Aspaveli (pegcetacoplan) continues to meet the criteria for the orphan drug designation status granted in 2017 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Orphan drug designation is granted to therapies that treat a serious disease that affects fewer than five in 10,000 people in the EU and provide a significant benefit over existing treatments. Aspaveli will have market exclusivity based on orphan drug designation for PNH.

Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.

Author: Rare Daily Staff

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