PTC’s Phase III Data Sets Up PKU Competition With BioMarin

 
• By Joseph Haas

Phase III data for PTC’s sepiapterin shows statistically significant reduction of blood phenylalanine levels in patients with phenylketonuria. BioMarin has the only two approved drugs for PKU.

Rare disease
PTC hopes to bring a competitor to BioMarin's PKU drugs to market • Source: Shutterstock

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