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Annexon Announces Clinical and Regulatory Progress for ANX005 Pivotal Program in Guillain-Barré Syndrome (GBS)
ANX005 Granted Orphan Drug Designation by the European Medicines Agency for the Treatment of GBS Target Enrollment Achieved in Phase 3 Pivotal Study of ANX005
About this update from Annexon, Inc.
[{"type":"text","content":"ANX005 Granted Orphan Drug Designation by the European Medicines Agency for the Treatment of GBS Target Enrollment Achieved in Phase 3 Pivotal Study of ANX005 in GBS Representing the Company’s Third Successfully Executed Clinical Study in GBS Company on Track to Report Pivotal Data in the First Half of 2024 BRISBANE, Calif., Oct. 10, 2023 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX) a clinical-stage biopharmaceutical company developing a new class of complement-based medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced that the European Medicines Agency (EMA) granted orphan drug designation to ANX005 for the treatment of Guillain-Barré Syndrome (GBS). The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to ANX005 for the treatment of GBS. ANX005, a humanized monoclonal antibody, inhibits C1q, the initiator molecule of the classical complement pathway, and is designed to stop complement mediated inflammation and neuronal damage early in GBS. The recent EMA orphan drug designation was based on an indirect comparison between ANX005 and intravenous immunoglobulin (IVIg) that demonstrated a notable and early improvement in muscle strength with ANX005 versus patients treated with IVIg, which translated into observable gains in health status, including a reduction in the need of mechanical ventilation. In granting the designation, EMA stated that preliminary clinical data with ANX005 constitutes “a clinically relevant advantage” over IVIg for patients affected by GBS. Importantly, Annexon has also achieved target enrollment of 225 patients in the randomized, double-blind, placebo-controlled Phase 3 trial of ANX005 in patients with GBS. This key milestone enables the company to deliver topline Phase 3 data in the first half of 2024. “GBS is a terrifying and underappreciated life-threatening condition that causes sudden onset of weakness in previously healthy people, leading to significant acute and long-lasting disability and, in some cases, death despite standard of care. This rare disease affects approximately 12,000 people in the U.S. and Europe each year, and the financial burden to the healthcare system is over $2 billion annually in the U.S. alone,” says Douglas Love, president and CEO of Annexon. “With European regul...