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PTC Therapeutics Announces FDA Target Regulatory Action Date for Sepiapterin and Lancet Publication of Phase 3 Trial Results
WARREN, N.J., Oct. 14, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has set a target regulatory action date of July
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"WARREN, N.J., Oct. 14, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has set a target regulatory action date of July 29, 2025, for the review of the New Drug Application (NDA) for sepiapterin, consistent with a standard review timeline. The NDA was submitted with an indication for the treatment of pediatric and adult patients with phenylketonuria (PKU), including all age groups and the full spectrum of disease subtypes. In addition, PTC announced that the results of the Phase 3 APHENITY trial were recently published in The Lancet, one of the most prestigious peer-reviewed medical journals.\n\n\"The Lancet publication reflects the promising and transformative nature of the data collected to date on sepiapterin and the therapy's potential to fill the persistent and significant unmet medical need for PKU patients,\" said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics.\nThe sepiapterin NDA includes the results from the Phase 3 APHENITY trial discussed in The Lancet publication, as well as data from the ongoing APHENITY open-label extension study, which provides evidence of the durability of effect of sepiapterin and the ability of patients to liberalize their diet and still maintain control of phenylalanine (Phe) levels. The most recent results from the Phe tolerance assessment demonstrate that approximately 60% of subjects achieve protein intake above the age-adjusted recommended daily allowance for an unaffected individual while still maintaining Phe levels \nAbout SepiapterinSepiapterin (formerly PTC923), an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase (PAH) enzyme. First, sepiapterin is a precursor compound that is rapidly absorbed and converted intracellularly to tetrahydrobiopterin (BH4), a critical cofactor of PAH. Sepiapterin also has an independent pharmacological chaperone effect, correcting PAH misfolding to enhance the enzyme function. Through this dual mechanism of action, sepiapterin effectively reduces blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients.\nAbout PhenylketonuriaPhenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed ...