- Review of European marketing application for PKU now initiated -
WARREN, N.J., May 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA.
"The validation of the MAA is an important step towards making sepiapterin available to children and adults affected by PKU in Europe," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The European submission is the first of several global submissions planned in 2024."
PTC expects to submit the sepiapterin NDA to the FDA no later than the third quarter of 2024. In addition, submissions are planned in a number of additional countries in 2024 including Brazil and Japan.
The sepiapterin MAA includes data from the phase 3 APHENITY trial which demonstrated a mean reduction in Phe levels of 63% in the overall treated population and 69% in the subgroup of subjects with classical PKU. The vast majority of subjects (84%) achieved Phe control in accordance with treatment guidelines of