UCB Secures FDA approval for Kygevvi in ultra-rare disease TK2d
UCB announced that Kygevvi (doxecitine and doxribtimine) has been granted approval by the US FDA in adults and paediatric patients living with thymidine kinase 2 deficiency (TK2d), with an age of symptom onset on or before 12 years. The approval comes in line with the company's guidance of a decision before YE25, making it the first and only approved treatment in this setting. Note that TK2d is an ultra-rare genetic mitochondrial disease with no approved therapies, and has a prevalence of 1.64 per 1m people. As such, we model peak sales of € 150m by 2034 and look forward to learn more from UCB about pricing as we approach commercialisation in 1Q26. € 270 TP and ACCUMULATE maintained.UCB is a global biopharmaceutical company focused on severe diseases in two therapeutic areas, namely central nervous system disorders and immunology. In the area of central nervous system disorders, Co. is focused on advanced Parkinson's disease, epilepsy, restless legs syndrome and epilepsy. In the area of immunology disorders, Co. is focused on rheumatoid arthritis, axial spondyloarthritis, psoriatic arthritis, systemic lupus erythematosus, juvenile idiopathic arthritis, post-menopausal osteoporosis, systemic lupus erythematosus and immunological diseases.
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