PTC Therapeutics, Inc. has announced its intention to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Upstaza. This gene therapy is being developed to combat aromatic L-amino acid decarboxylase deficiency. In addition, following recent interactions with the FDA, PTC intends to reapply for a New Drug Application (NDA) for Translarna, a treatment for nonsense mutation Duchenne muscular dystrophy. The company aims to re-submit this NDA by the middle of the year.
Furthermore, PTC Therapeutics confirmed that the submission of the sepiapterin Marketing Authorization Application (MAA) for Phenylketonuria (PKU) to the European Medicines Agency (EMA) is still on track to be submitted by the end of the month.
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