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PTC Therapeutics Announces Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) in Japan
– Indication includes all ages and the full spectrum of disease severity –– First Japan product approval for PTC – WARREN, N.J., Dec. 22, 2025 /PRNewswire/ --
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"– Indication includes all ages and the full spectrum of disease severity –– First Japan product approval for PTC – WARREN, N.J., Dec. 22, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that Sephience™ (sepiapterin) has been approved by the Japanese Ministry of Health, Labor and Welfare (MHLW) for the treatment of children and adults living with phenylketonuria (PKU). The label includes individuals of all ages and the full spectrum of disease severity.\n \n \n \n \n \n \n \n\"The approval of Sephience in Japan is an important milestone for Japanese patients living with PKU as well as for PTC,\" said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. \"I am proud that we have been able to achieve approvals for Sephience in the EU, US and Japan within less than six months, allowing for a true global launch. Our team in Japan is fully built and excited to bring Sephience to any individual who may benefit.\"PTC will now engage in pricing discussions which are expected to conclude in Q1 2026, with launch occurring shortly thereafter. About Sephience™ (sepiapterin)Sephience™ is indicated for the treatment of adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients. Sephience is approved in the European Union / European Economic Area region, the United States and Japan.About PhenylketonuriaPhenylketonuria (PKU) is a rare, inherited metabolic disease, characterized by the body's inability to break down an essential amino acid called phenylalanine (Phe) and which can result in neurological and other symptoms. If left untreated or poorly managed, Phe can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with PKU initially do not have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible. Diagnosis of PKU usually takes place during newborn screening progr...