– Indication includes all ages and the full spectrum of disease severity –
– First Japan product approval for PTC –

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.

"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.