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›Vaccins et laboratoires de recherche
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Context
Period
Period: S1 2026
Key reported figures
Revenue: 4 270 millions d'euros
Quarterly revenue: 4 270 millions d'euros
Revenue growth: 22,5 %
EBITDA: 1 685 millions d'euros
EBITDA margin: 40,7 %
Net income: 1 111 millions d'euros
2 821 millions d'euros
Guidance from the release
Based on the continued strong growth, revenue is expected to grow in the low-teens to mid-teens percentage range; Reflecting our strong execution and confidence in the outlook
Risks mentioned
Perte d'exclusivité de BRIVIACT aux États-Unis depuis février 2026 et en Europe attendue en août 2026
Ventes de VIMPAT au Japon affectées par l'arrivée de génériques fin 2025
Expiration en 2026 de la protection brevetaire de CIMZIA au Japon
Opportunities identified
Objectif de ventes maximales de BIMZELX relevé à au moins 7 milliards d'euros
Supériorité de BIMZELX sur risankizumab dans le rhumatisme psoriasique (étude BE BOLD)
FINTEPLA déposé dans le syndrome CDKL5 auprès de la FDA et de l'EMA, Phase 3 lancée dans le syndrome de Rett
Outlook / guidance
Management commentary: revenue is expected to grow in the low-teens to mid-teens percentage range at constant exchange rates (CER). Adjusted EBITDA² is expected to grow in the mid-teens to low-twenties percentage range at CER. Excluding other operating one-offs in 2025, adjusted EBITDA² growth is expected to be in the mid-twenties to low-thirties percentage range at CER.
The information presented in this article is provided for informational purposes only and does not constitute an investment recommendation, an incentive to buy or sell a financial asset, or investment advice. Readers are invited to conduct their own research before making any decision.
Investments in the stock market involve risks, including the risk of capital loss. Past performance of an asset or market is no guarantee of future results. Any investment decision should be made taking into account your personal financial situation, objectives and risk tolerance.
