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APPLY-PNH Final 48-Wk Results: Phase III Trial of Iptacopan Monotherapy in Patients With PNH and Residual Anemia With Anti-C5 Therapy

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Among patients with PNH and persistent anemia despite anti-C5 therapy, 48-week data from the APPLY-PNH trial show that iptacopan monotherapy yields durable hematologic and clinical improvements and comprehensive control of intravascular and extravascular hemolysis, supporting iptacopan as a novel practice-changing treatment option.

Released: December 14, 2023


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Provided by Clinical Care Options, LLC

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Supported by educational grants from AbbVie Inc., AstraZeneca, Daiichi Sankyo, Inc., Merck Sharp & Dohme LLC, Novartis Pharmaceuticals Corporation, Regeneron Pharmaceuticals, Inc, and Seagen Inc.

AbbVie Inc.


Daiichi Sankyo, Inc.

Merck Sharp & Dohme, LLC

Novartis Pharmaceuticals Corporation

Regeneron Pharmaceuticals, Inc

Seagen Inc.