Drug Profile

Rivunatpagene miziparvovec - Ultragenyx Pharmaceutical

Alternative Names: UX-701

Latest Information Update: 09 Oct 2024

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At a glance

Originator Ultragenyx Pharmaceutical
Class Gene therapies
Mechanism of Action Copper-transporting ATPase replacements; Gene transference

Orphan Drug Status

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

Yes - Hepatolenticular degeneration
New Molecular Entity No
Available For Licensing Yes

Highest Development Phases

Phase I/II Hepatolenticular degeneration

Most Recent Events

09 Oct 2024 Efficacy and adverse events data from a phase I/II/III Cyprus²⁺ trial in Hepatolenticular degeneration released by Ultragenyx Pharmaceutical
16 Oct 2023 Efficacy and adverse events data from a phase I/II/III trial in Hepatolenticular degeneration released by Ultragenyx Pharmaceutical
31 Dec 2022 Ultragenyx Pharmaceutical has patent protection for rivunatpagene miziparvovec

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