PTC Therapeutics Gets Marketing Authorization For Sephience In EU To Treat Phenylketonuria

(RTTNews) - PTC Therapeutics, Inc. (PTCT) Monday said that the European Commission has granted marketing authorization for Sephience for the treatment of phenylketonuria (PKU).

The company noted that the uthorization includes a broad label inclusive of all ages and disease severities.

The approval is based on results from the Phase 3 APHENITY study 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.

The European launch of Sephience will be initiated in Germany in the first half of July.

The company has submitted a new drug application (NDA) for Sephience to the FDA, which the regulator is expected to decide on July 29 this year. The drug is under review in several other countries including Japan and Brazil.