- In January 2024, Jnana Therapeutics announced positive clinical proof of concept for JNT-517, a potential first-in-class oral treatment for phenylketonuria (PKU). This treatment is designed to address the underlying metabolic deficiency in PKU by enabling the body to break down phenylalanine, the amino acid that accumulates in patients with the disorder. The successful study results highlight JNT-517’s potential to offer a new therapeutic option for PKU patients, providing an alternative to strict dietary management. If approved, this oral treatment could improve the quality of life for individuals living with PKU by reducing reliance on dietary restrictions
- In May 2024, PTC Therapeutics announced that the European Medicines Agency (EMA) will review its Marketing Authorization Application (MAA) for Sepiapterin, a treatment for phenylketonuria (PKU). Sepiapterin is designed to reduce the levels of phenylalanine in PKU patients by enhancing the body’s ability to produce the necessary enzyme to metabolize it. If approved, this therapy would provide a novel option for PKU patients, offering a potential treatment to complement dietary restrictions and improve overall management of the condition. The EMA’s review is a critical step toward bringing this innovative treatment to the market
