PTC Therapeutics, Inc. (NASDAQ: PTCT) has reached a pivotal milestone with the FDA's acceptance for filing of the New Drug Application (NDA) for sepiapterin, targeting the treatment of both pediatric and adult patients suffering from phenylketonuria (PKU). The announcement was made on October 1, 2024, highlighting the potential of sepiapterin to significantly benefit PKU patients by enabling diet liberalization and reducing phenylalanine (Phe) levels.
Clinical Trial Data: The NDA submission is backed by the results from the phase 3 APHENITY trial, which showed a mean reduction in Phe levels of 63% in the overall treated population and 69% in the subgroup with classical PKU. Notably, 84% of subjects achieved Phe control in line with treatment guidelines, underscoring the therapy's effectiveness.
Community Impact: Matthew B. Klein, M.D., CEO of PTC Therapeutics, emphasized the importance of this development for the PKU community, stating it as a critical step towards addressing the significant unmet needs of patients. Catherine Warren, Executive Director of the National PKU Alliance, also expressed excitement over the progress, highlighting the potential for sepiapterin to lessen the dependency on restrictive diets, thereby improving the quality of life for individuals with PKU.
This advancement represents a hopeful future for PKU patients, offering a new avenue for managing their condition more effectively.
