Drug Profile

Vemircopan - Alexion AstraZeneca Rare Disease

Alternative Names: ACH 5228; ACH-0145228; ALXN 2050

Latest Information Update: 26 Feb 2025

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At a glance

Originator Achillion Pharmaceuticals
Developer Alexion AstraZeneca Rare Disease
Class Amides; Azabicyclo compounds; Halogenated hydrocarbons; Immunotherapies; Indazoles; Pyrimidines; Small molecules; Urologics
Mechanism of Action Complement factor D inhibitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Paroxysmal nocturnal haemoglobinuria
New Molecular Entity Yes

Phase I Autoimmune disorders
Discontinued IgA nephropathy; Lupus nephritis; Myasthenia gravis; Paroxysmal nocturnal haemoglobinuria

06 Feb 2025 Discontinued - Phase-II for IgA nephropathy in United Kingdom, Turkey, Thailand, Taiwan, Spain, Peru, Mexico, South Korea, Italy, Israel, Germany, China, Brazil, Australia, Argentina, USA (PO) before February 2025 (AstraZeneca pipeline, February 2025)
06 Feb 2025 Discontinued - Phase-II for Lupus nephritis in United Kingdom, Turkey, Thailand, Taiwan, Spain, Serbia, Peru, Mexico, South Korea, Italy, Israel, Germany, China, Australia, Brazil, Argentina, USA (PO) before February 2025 (AstraZeneca pipeline, February 2025)
09 Dec 2024 Alexion AstraZeneca Rare Disease terminates phase II trial in IgA nephropathy and Lupus nephritis in USA, United Kingdom, Turkey, Thailand, Taiwan, Spain, Peru, Mexico, South Korea, Italy, Israel, Germany, China, Brazil, Australia, Argentina (PO) (NCT05097989)

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