Doxecitine and Doxribtimine: First Approval

Pediatric Drugs 2026 Open access

Read full text
Authors: Hannah A. Blair
DOI: 10.1007/s40272-025-00734-1
Cited by: 4
References: 7

Abstract

) are pyrimidine nucleosides developed by UCB for the treatment of thymidine kinase 2 deficiency (TK2d). In November 2025, doxecitine and doxribtimine received its first approval in the USA for the treatment of TK2d in adult and pediatric patients with an age of symptom onset on or before 12 years. This article summarizes the milestones in the development of doxecitine and doxribtimine leading to this first approval for TK2d.

Fields of Study & Subjects

Chronic Lymphocytic Leukemia Research Immunodeficiency and Autoimmune Disorders Genetics and Neurodevelopmental Disorders

Publication details

Journal / source
Pediatric Drugs
Publisher
Springer Science and Business Media LLC
Volume
28
Issue
2
Pages
209–215
Publication date
28 January 2026
Language
EN