Aspaveli® (pegcetacoplan) approved in Europe for treatment-naïve adults with PNH

STOCKHOLM, May 8, 2024 -- Sobi® has announced that the European Commission (EC) has approved an extension of the indication for Aspaveli® (pegcetacoplan) to treat adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who suffer from haemolytic anaemia. Previously, Aspaveli was approved in Europe for adults with PNH who remained anaemic after receiving a C5 inhibitor for at least three months. With this new approval, Aspaveli becomes the first C3 inhibitor authorized for first-line treatment of PNH in Europe, offering improved outcomes by enhancing haemoglobin levels and other clinical indicators through its unique mechanism of action.

Lydia Abad-Franch MD, the Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi, highlighted the significance of this approval. "Today's announcement validates the strong clinical data backing Aspaveli's efficacy and safety, providing healthcare professionals and patients with enhanced tools to manage PNH effectively," she stated. "European patients now have the option to start treatment with Aspaveli from diagnosis or switch from their existing C5 inhibitor therapy if they exhibit signs of haemolytic anaemia. This milestone underscores our commitment to improving treatment choices for those impacted by this rare and complex disorder."

PNH is a chronic, life-threatening blood disorder characterized by the destruction of red blood cells due to uncontrolled complement activation. Symptoms include persistently low haemoglobin levels, frequent need for transfusions, and severe fatigue caused by anaemia. Despite advancements with C5 inhibitor treatments, about 86% of people treated with these inhibitors remain anaemic, according to surveys conducted in the US and EU.

The extension of the indication is based on findings from the APL2-308 (PRINCE, NCT04085601) study. This open-label, randomized, comparator-controlled study involved patients with PNH who had not received any complement inhibitor treatment within three months before enrollment and had haemoglobin levels below normal and lactate dehydrogenase levels at least 1.5 times the upper limit of normal. The efficacy and safety of Aspaveli were assessed over 26 weeks compared to standard care, which included transfusions, corticosteroids, and supplements like iron, folate, and vitamin B12, excluding complement inhibitors.

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

Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 therapy designed to control excessive activation of the complement system, which can trigger the progression of various serious diseases. Pegcetacoplan is approved for treating PNH in the European Union, the United States, and other global markets. It is also being investigated for other rare diseases in haematology and nephrology.

Sobi and Apellis collaborate globally on the development of systemic pegcetacoplan. Sobi has exclusive commercialization rights outside the US for systemic pegcetacoplan, while Apellis retains US exclusive commercialization rights and worldwide rights for ophthalmological applications of pegcetacoplan, including geographic atrophy (GA).

Sobi® is an international biopharmaceutical company dedicated to transforming the lives of individuals with rare and debilitating diseases. Specializing in haematology, immunology, and specialty care, Sobi employs roughly 1,800 people across Europe, North America, the Middle East, Asia, and Australia. In 2023, the company reported revenue of SEK 22.1 billion and is listed on Nasdaq Stockholm under the share (STO:SOBI).