Shots:
- Health Canada has approved PTC‘s Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in pts of all ages, with broad labeling for hyperphenylalaninemia in pts (≥1mos.) with sepiapterin-responsive PKU
- Approval was based on APHENITY study results & long-term extension data showing durable effects & favorable safety in PKU pts
- Sephience is a natural precursor of the enzymatic co-factor BH4 that boosts PAH enzyme activity & lowers blood phenylalanine levels to treat a broad range of PKU pts
Ref: CA Newswire | Image: PTC Therapeutics |Press Release
Related News: PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria
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