Drug Profile

UX 055

Alternative Names: UX-055

Latest Information Update: 13 Jan 2023

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At a glance

Originator Ultragenyx Pharmaceutical
Class Antiepileptic drugs; Gene therapies
Mechanism of Action Cyclin-dependent kinase 5 replacements; Gene transference

Orphan Drug Status

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

No
New Molecular Entity No

20 Dec 2022 Ultragenyx plans a non interventional trial for Early-infantile-epileptic-encephalopathy-2 in USA (Ultragenyx pharmaceuticals pipeline, December 2022)
05 Dec 2022 Preclinical trial in Early infantile epileptic encephalopathy 2 in USA (Intrathecal), before December 2022 (Ultragenyx pharmaceuticals pipeline, December 2022)
11 Feb 2021 Ultragenyx pharmaceuticals files a Patent Cooperation Treaty application for UX 055

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