PTC Therapeutics receives FDA approval for Sephience (sepiapterin) to treat PKU in patients 1 month and older

PTC THERAPEUTICS, INC.

FDA approved Sephience for hyperphenylalaninemia in adult/pediatric patients ≥1 month with sepiapterin-responsive PKU.
Approval based on Phase 3 APHENITY trial and long-term extension data showing significant efficacy and safety.
Broad labeling includes all disease subtypes; PTC CEO states Sephience could become standard of care.
SEPHIENCE recently received EU marketing authorization; reviews ongoing in Japan, Brazil and other countries.
PTC to host conference call July 28 at 5pm ET to discuss approval.

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