FDA green lights broader label for PTC Thera’s Sephience for PKU
US biotech PTC Therapeutics’ (Nasdaq: PTCT) shares trading up 4.8% to $46.48 in after-hours activity yesterday, as it announced that the US Food and Drug Administration (FDA) has approved Sephience (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU).
The approval, which comes just a month after a similar authorization from the European Commission, includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients one month of age and older with sepiapterin-responsive PKU.
The global phenylketonuria treatment market is a growing market, with a projected value of $1.62 billion by the end of 2034. The market was valued at $905.71 million in 2024, driven by rising incidence and advancements in treatment.
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