UCB: Positive Opinion for KYGEVVI in the Treatment of TK2 Deficiency
UCB announces that a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommends granting marketing authorization for KYGEVVI (doxecitine and doxribtimide) in the treatment of thymidine kinase 2 deficiency (TK2d). This decision potentially paves the way for the first and only approved therapeutic option in the European Union for this ultra-rare disease.
Positive CHMP Opinion for Market Authorization
The CHMP has adopted a positive opinion recommending the granting of marketing authorization under exceptional circumstances for KYGEVVI (doxecitine and doxribtimide) for pediatric and adult patients with genetically confirmed thymidine kinase 2 deficiency, with symptom onset at or before the age of 12 years. If approved by the European Commission, KYGEVVI would be the first and only approved treatment in the European Union indicated for this condition. The final decision of the European Commission is expected in the second quarter of 2026. The drug has benefited from the EMA's PRIME (PRIority MEdicines) program, which provides enhanced scientific and regulatory support for medicines addressing unmet medical needs.
Supporting Data from Clinical Studies
The data supporting this opinion come from two studies involving 39 participants suffering from TK2d with symptom onset at or before the age of 12 years. These studies investigated the impact of the treatment on functional outcomes (motor milestones, ventilatory support, nutritional support) as well as survival. The drug functions by being incorporated into the mitochondrial DNA of skeletal muscle, restoring the number of mitochondrial DNA copies and improving muscle function. The most common adverse effects associated with the treatment were gastrointestinal disorders, including diarrhea, vomiting, and abdominal pain.
