• UCB Receives Positive EU Recommendation for First-Ever Thymidine Kinase 2 Deficiency Treatment
BRUSSELS, Belgium – Global biopharmaceutical company UCB announced on January 30 that a European Medicines Agency committee has recommended its drug KYGEVVI for approval.
If authorized, it would become the first treatment in the European Union for Thymidine Kinase 2 Deficiency, an ultra-rare genetic disease.
What is new?
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for KYGEVVI®, a combination of doxecitine and doxribtimine. The recommendation is for the treatment of pediatric and adult patients with genetically confirmed thymidine kinase 2 deficiency (TK2d) who showed symptoms on or before the age of 12. The final decision from the European Commission is anticipated in the second quarter of 2026. If approved, KYGEVVI® would be the only treatment indicated for this condition in the EU.
About the Disease
Thymidine kinase 2 deficiency (TK2d) is an ultra-rare, life-threatening genetic mitochondrial disease. It is characterized by progressive and severe muscle weakness, known as myopathy. The condition can affect a person’s ability to walk, eat, and breathe independently, often requiring continuous caregiver support. There are currently no approved treatment options in Europe beyond supportive care. The disease is often fatal, particularly when symptoms begin on or before age 12, with a high risk of premature death. The estimated worldwide prevalence is 1.64 cases per 1,000,000 people.
Regulatory and Clinical Context
The CHMP recommendation is for a marketing authorization under “exceptional circumstances,” a pathway used for rare diseases where providing comprehensive efficacy and safety data is not possible or is unethical. The drug was also supported through the EMA’s PRIority MEdicines (PRIME) scheme, which provides enhanced regulatory support for medicines addressing unmet medical needs. Supportive data for the recommendation came from two studies of 39 patients. Doxecitine and doxribtimine work by being incorporated into mitochondrial DNA, restoring its copy number and improving skeletal muscle function. The most common side effects observed in the studies were gastrointestinal disorders, including diarrhoea, vomiting, and abdominal pain.
About this company
UCB is a global biopharmaceutical company based in Brussels, Belgium, focused on discovering and developing medicines for severe diseases of the immune system and the central nervous system. The company employs more than 9,000 people in approximately 40 countries and generated revenue of €6.1 billion in 2024. UCB is listed on the Euronext Brussels stock exchange under the symbol UCB.
Read more
UCB Raises 2025 Revenue Forecast, Citing Strong BIMZELX Performance
UCB Epilepsy Drug Fenfluramine Shows Positive Phase 3 Results
Update: UCB’s Bimzelx Approved By FDA, Offering 8-Week Dosing