Drug Profile

VX 864

Alternative Names: VX-864

Latest Information Update: 06 Oct 2024

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At a glance

Originator Vertex Pharmaceuticals
Class Small molecules
Mechanism of Action Alpha 1-antitrypsin modulators

Orphan Drug Status

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

No
New Molecular Entity Yes

Highest Development Phases

Discontinued Alpha 1-antitrypsin deficiency

Most Recent Events

19 Aug 2024 Vertex Pharmaceuticals completes a phase II trial in Alpha 1-antitrypsin deficiency in USA, Germany, Ireland and the United Kingdom (PO)(NCT05643495) (EudraCT2022-002746-40)
06 Nov 2023 Discontinued - Phase-II for Alpha 1-antitrypsin deficiency in USA (PO) due to non-serious rash events in some patients
04 Jan 2023 VX 864 is still in phase II trials for Alpha 1-antitrypsin deficiency in USA (PO) (Vertex Pharmaceuticals pipeline, January 2023)

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