#United States #PTC Therapeutics #Warren #Sephience #PKU Treatment

PTC Therapeutics Plans Launch of Sephience for PKU Treatment

PTC Therapeutics, Inc. (NASDAQ: PTCT) has received a significant boost in its mission to address the needs of individuals living with phenylketonuria (PKU) following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency. This decision marks a pivotal moment in the company's journey towards making Sephience™ (sepiapterin) widely available for patients across Europe.

The CHMP's favorable opinion is groundbreaking, as it encompasses a broad label for Sephience, which will cater to patients of all ages and with varying degrees of severity concerning PKU. The CEO of PTC Therapeutics, Dr. Matthew B. Klein, expressed his enthusiasm, stating that this positive development brings hope to the PKU community in Europe, where there remains an important gap in medical treatment options.

Sephience is designed to treat both children and adults suffering from PKU, a rare metabolic disorder that can lead to severe cognitive and developmental impairments if not managed effectively. The innovative treatment functions through a dual mechanism, improving the function of the phenylalanine hydroxylase (PAH) enzyme, which is crucial in breaking down phenylalanine (Phe)—an amino acid that, when accumulated, can become toxic. Through its ability to lower blood Phe levels and facilitate dietary liberalization, Sephience promises to significantly enhance the quality of life for PKU patients, even those facing the most challenging forms of the condition.

As preparations for the European launch are already in motion, PTC is focusing initial efforts on Germany and other pivotal markets. The company expects that named patient access will be available immediately following the formal adoption of the CHMP opinion by the European Commission, anticipated to occur within the next two months. This decision will not only affect Germany but will be valid across all 27 EU member states as well as Iceland, Norway, and Liechtenstein, providing a wider safety net for PKU patients.

Simultaneously, PTC is also awaiting a crucial New Drug Application (NDA) review from the U.S. Food and Drug Administration (FDA) for sepiapterin, with a target action date set for July 29, 2025. The company is working diligently towards securing regulatory approvals in other global markets, including Brazil and Japan, where the unmet need for effective PKU treatments is substantial.

Understanding PKU

Phenylketonuria, or PKU, is an inherited metabolic disorder resulting from a defect in the gene responsible for producing the PAH enzyme. When untreated, the elevated levels of Phe can lead to irreversible neurological damage, including developmental delays and intellectual disabilities. In most cases, PKU is detected through newborn screening programs, enabling timely interventions. It is estimated that around 58,000 individuals worldwide are living with PKU, highlighting the urgent need for comprehensive and effective treatment approaches.

About PTC Therapeutics

PTC Therapeutics is committed to improving the lives of individuals with rare disorders through innovative therapeutic solutions. Their focus on developing and commercializing advanced medicines that meet significant unmet medical needs underlines the importance they place on research and development in the biopharmaceutical landscape. For further details and updates on PTC's initiatives, the company encourages everyone to visit their website or engage with them on social media platforms.

As the landscape of treatment for PKU evolves with the potential approval of Sephience, the PTC Therapeutics team continues its dedication to addressing the needs of its patients, moving towards a brighter future for those affected by PKU.