UCB’s Kygevvi (doxecitine and doxribtimine) has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending marketing authorisation under exceptional circumstances. 

The recommendation is 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. 

“This positive CHMP opinion marks a turning point in the treatment of TK2d, offering a new chapter of hope and a meaningful step forward for patients, families, and clinicians alike,” said Donatello Crocetta, Chief Medical Officer at UCB. “We are deeply proud to be one step closer to being able to bring the first and only approved treatment for TK2d to the community.” 

Doxecitine and doxribtimine was supported through EMA’s PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a potential to address patients’ unmet medical needs.  

Marketing authorisation under exceptional circumstances may be granted to medicines where the applicant is unable to provide comprehensive data on efficacy and safety under normal conditions of use, because the condition to be treated is rare or because collection of full information is not possible or is unethical.  

“Doxecitine and doxribtimine treatment is a great example of how robust pre-clinical data can be translated into a clinical programme in ultra-rare disease, impacting the natural history of a fatal disorder such as thymidine kinase 2 deficiency,” said Caterina Garone, Associate Professor of Medical Genetics, University of Bologna, Italy. “The CHMP positive opinion is a significant step toward potential access to the first treatment option for TK2d in Europe.” 

TK2d is an ultra-rare, life-threatening, genetic mitochondrial disease characterised by progressive and severe myopathy with no approved treatment options in Europe.