Doxecitine + Doxribtimine 2g+2g Suppliers & Bulk Manufacturers

Available Forms: Oral Solution

Available Strengths: 2g+2g

Reference Brands: Kygevvi (USA)

Category: Orphan Drugs

Doxecitine (2′‑deoxycytidine) and doxribtimine (2′‑deoxythymidine) provide nucleosides to restore mitochondrial DNA synthesis in patients with TK2 deficiency. This helps improve mitochondrial function in muscle cells, reduce muscle weakness, delay disease progression, and improve survival.

Product Description:

This oral solution supplies two deoxynucleosides — doxecitine, which is deoxycytidine, and doxribtimine, which is deoxythymidine. In thymidine kinase 2 deficiency, a mitochondrial disease, cells cannot phosphorylate these building blocks, so mitochondria run short of the nucleotides they need to copy their own DNA. Mitochondrial DNA is progressively depleted, and muscle, which depends heavily on mitochondrial energy, fails. Supplying the substrates in large amounts allows an alternative salvage route to bypass the missing enzyme.

It is used for thymidine kinase 2 deficiency in adults and children, and it is the first and only approved treatment for the condition. The disease often begins in infancy with progressive weakness, loss of motor milestones and respiratory failure; in trials, treated patients showed improved survival and motor function compared with untreated natural history. It is taken several times daily, mixed in water. Diarrhoea is the most common adverse effect.

Sourcing notes. Originator orphan product approved in the US in 2025; registration elsewhere is not yet established, so supply outside the US runs through named-patient or expanded access routes. Genetic confirmation of the diagnosis is required. Metabolic and neuromuscular centres are the point of care.