EC approves PTC Thera’s Sephience for PKU

US biotech PTC Therapeutics’ (Nasdaq: PTCT) Sephience (sepiapterin) has been granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and disease severities.

"The European approval of Sephience is a great step in our efforts to bring this safe and highly effective therapy to children and adults affected by PKU worldwide," said Dr Matthew Klein, chief executive of PTC Therapeutics. "The broad label supports that potential for Sephience to address all key PKU patient segments and become the new standard of care," he added.

The European approval is based on the highly statistically significant results from the
Phase III APHENITY trial as well as evidence of durable treatment effect and the ability of study participants to liberalize their diet in the APHENITY long-term extension study.