FDA approves 1st drug for thymidine kinase 2 deficiency

The US Food and Drug Administration (FDA) has approved Belgium drugmaker UCB’s (Euronext: UCB) Kygevvi (doxecitine and doxribtimine) powder to treat thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients who start to show symptoms when they are 12 years old or younger.

Kygevvi received Breakthrough Therapy designation for this indication, and made it the first and only approved treatment for patients living with TK2d.

Due to a lack of drugs, the market is small, estimated at just around $1 million in 2023, with the US holding the largest share. However, a recent forecast projects the global market could reach $3.74 billion by 2032.