UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that the European Commission (EC) has granted marketing authorization under exceptional circumstances for KYGEVVI (doxecitine and doxribtimine) for the treatment of paediatric and adult patients with genetically confirmed thymidine kinase 2 deficiency (TK2d) with an age of symptom onset on or before 12 years.1 It is the first and only approved treatment for TK2d.

FDA approves 1st drug for thymidine kinase 2 deficiency, a very rare mitochondrial disease

This novel therapy offers the first FDA-approved option to address the root cause of this ultra-rare condition, which causes progressive muscle weakness...

The European Commission has approved doxecitine and doxribtimine, the first therapy for pediatric and adult patients with TK2d.

The combination therapy doxecitine and doxribtimine (Kygevvi®) has been approved by the US Food and Drug Administration (FDA) as the first and only

UCB's Kygevvi becomes the first FDA-approved treatment for thymidine kinase 2 deficiency, offering hope to patients with this rare neuromuscular disorder.

FDA approves 1st drug for thymidine kinase 2 deficiency, a very rare mitochondrial disease

Doxecitine and doxribtimine powder given a thumbs up for thymidine kinase 2 deficiency

The FDA has approved Kygevvi (doxycitine and doxribtimine), the first therapy to improve functional outcomes and survival in thymidine kinase 2 deficiency.

STELLANEWS.LIFE（ステラニュース・ライフ）は、科学や技術、医薬品分野における最新の研究成果や発見 | STELLANEWS.LIFE（ステラニュース・ライフ）は、科学や技術、医薬品分野における最新の研究成果や発見

Data indicate that KYGEVVI therapy improved survival and functional outcomes in people with TK2d.1 Additional data from the largest pool of information on TK2d illustrates the impact and burden of disease progression2 and highlights the benefits of early diagnosis.3 ATLANTA, June 17, 2026...

Clinical data highlight survival benefits and improvement in functional motor outcomes associated with treatment with doxecitine (dC) and doxribtimine (dT) - an investigational pyrimidine nucleoside therapy - in people living with thymidine kinase 2…