Last reviewed · How we verify
Kygevvi (DOXRIBTIMINE)
Kygevvi works by replacing the deficient enzyme thymidine kinase 2 in patients with TK2d.
At a glance
| Generic name | DOXRIBTIMINE |
|---|---|
| Sponsor | Ucb Inc |
| Modality | Small molecule |
| Therapeutic area | Other |
| Phase | FDA-approved |
| First approval | 2025 |
Mechanism of action
Administration of KYGEVVI is intended to incorporate the pyrimidine nucleosides, deoxycytidine and deoxythymidine, into skeletal muscle mitochondrial deoxyribonucleic acid (DNA). This action restores mitochondrial DNA copy number in TK2d mutant mice.
Approved indications
- Thymidine Kinase 2 Deficiency
Common side effects
- Diarrhea
- Abdominal pain (including abdominal pain upper)
- Vomiting
- Alanine aminotransferase increased (ALT)
- Aspartate aminotransferase increased (AST)
- Elevated liver enzymes
- Elevated liver transaminases
- Elevated AST/ALT at baseline
- Last post-baseline ALT values higher severity than baseline
Key clinical trials
- A RETROspective Study of Patients With TK2d
- A Retrospective Study of Subjects With Thymidine Kinase 2 Deficiency
- An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency (PHASE2)
- Doxecitine and Doxribtimine-Expanded Access
- Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) Deficiency (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |