Pharma Focus Europe

Aspaveli® (pegcetacoplan) Gets Green Light in Europe for Treatment-Naïve Adult Patients with PNH

Thursday, May 09, 2024

Sobi® has announced that the European Commission (EC) has granted approval for an expanded indication for Aspaveli® (pegcetacoplan) in treating adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who suffer from haemolytic anaemia.

Aspaveli is already approved in Europe for treating anaemic adults with PNH who have undergone treatment with a C5 inhibitor for at least three months. This recent approval makes Aspaveli the first C3 inhibitor endorsed for first-line treatment of PNH in Europe, promising effective outcomes by enhancing haemoglobin levels and other clinical markers due to its distinct mode of action.

Sobi, stated, "Today's approval highlights the robust clinical evidence supporting Aspaveli's effectiveness and safety, providing healthcare professionals and patients with an expanded arsenal for managing PNH. European patients can now begin treatment with Aspaveli upon diagnosis or transition from their current C5 inhibitor therapy if they exhibit signs of haemolytic anaemia. This significant milestone reflects our commitment to enhancing treatment options for those affected by this rare and complex condition."

PNH is a rare, chronic, and life-threatening blood disorder characterized by uncontrolled complement activation, leading to the destruction of oxygen-carrying red blood cells. Marked by persistently low haemoglobin levels, PNH can necessitate frequent transfusions and cause debilitating symptoms such as severe fatigue from anaemia. Despite advancements with C5 inhibitor treatment, up to 86 percent of individuals with PNH treated with C5 inhibitors remain anaemic, according to cross-sectional surveys conducted in the US and EU.

The indication extension is supported by data from APL2-308 (PRINCE, NCT04085601), an open-label, randomized, comparator-controlled study that enrolled PNH patients who had not received any complement inhibitor treatment within three months prior to enrollment and had haemoglobin levels below the lower limit of normal and lactate dehydrogenase levels ≥1.5 times the upper limit of normal. The efficacy and safety of Aspaveli were assessed over a 26-week duration compared to standard of care, excluding complement inhibitors.

Aspaveli is the European trade name for pegcetacoplan, also known as Empaveli® in the United States, where it is approved for treating adults with PNH and marketed by Apellis.

Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is approved for treating paroxysmal nocturnal haemoglobinuria (PNH) as Aspaveli/Empaveli in the European Union, United States, and other countries worldwide. Aspaveli/Empaveli is also being investigated for other rare diseases in hematology and nephrology.



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