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.
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
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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.
SectorPharmacie et biotechnologies›Pharmacie, vaccins et santé animale
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Context
Period
Period: FY2026
Guidance from the release
higher 2026 expectations, with revenue growth anticipated in the low twenties
Risks mentioned
Marché des anti-TNF en contraction : CIMZIA y progresse encore en volume, mais sur un marché qui recule.
Opportunities identified
BIMZELX plus fort aux États-Unis : demande des patients soutenue et prix nets plus favorables.
CIMZIA : contribution solide, croissance des volumes et prix plus favorables.
Effet de levier opérationnel : la croissance plus forte du chiffre d'affaires élargit nettement la marge.
Outlook / guidance
Management commentary: Continued strong performance supports higher 2026 expectations, with revenue growth anticipated in the low twenties at constant exchange rates, up from low- to mid-teens Higher revenue growth is driving significant operating leverage, with adjusted EBITDA growth expected in the low- to mid-forties at constant exchange rates, up from mid-teens to low-twenties
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