Skip to content Skip to footer
Subscribe Now
NEWS

PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria

Shots:The US FDA has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in pts of all ages, with broad labeling for hyperphenylalaninemia in adults & pediatric pts (≥1mos.) with sepiapterin-responsive PKU; review is ongoing in Japan & BrazilApproval was based on APHENITY study results & long-term extension data showing durable effects and…
July 29, 2025

Read more

PTC Therapeutics
NEWS

PTC Therapeutics’ Sephience Receives the CHMP’s Positive Opinion for the Treatment of Phenylketonuria (PKU) 

Shots: The CHMP has issued a positive opinion recommending marketing authorization for Sephience (sepiapterin) to treat PKU in patients of all ages and disease severities, expected the EC approval within two mos., with the decision applying across the EU including Iceland, Norway, and Liechtenstein Sepiapterin's NDA is under FDA review (PDUFA: July 29, 2025), and…
April 25, 2025

Read more