PNH is a rare, chronic, life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells through intravascular and extravascular hemolysis. Characterized by persistently low hemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue. Despite improvements in hemolytic activity with C5 inhibitor treatment, approximately 72% of C5 treated patients remain anemic, according to a retrospective and a cross-sectional study.1,2
'Building on our recent
The positive opinion is based on the results from the head-to-head Phase 3 PEGASUS 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 results were published in the
'Today's positive opinion by the CHMP is a significant milestone for people living with PNH across
Aspaveli is the European trade name for pegcetacoplan, which is known as EMPAVELI in
About the PEGASUS Study
PEGASUS (APL2-302; NCT03500549) was a multi-center, randomized, head-to-head Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of Aspaveli/EMPAVELI (pegcetacoplan) compared to eculizumab.
About Aspaveli/EMPAVELI (pegcetacoplan)
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. EMPAVELI is approved in
About Apellis
About Sobi
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across
Apellis Forward-Looking Statement
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute 'forward-looking statements' within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the implications of clinical data. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether pegcetacoplan will receive approval from the EMA for PNH when expected or at all and other factors discussed in the 'Risk Factors' section of Apellis' Quarterly Report on Form 10-Q filed with the
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