European Commission Approves Sephience™ for PKU Treatment
Sephience™ (sepiapterin) Granted Marketing Authorization by the European Commission
In a noteworthy development, PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that Sephience™ (sepiapterin) has been granted marketing authorization by the European Commission for treating individuals with phenylketonuria (PKU), a metabolic disorder affecting both children and adults. This authorization signifies a major advancement in therapeutic options available for PKU patients, making it inclusive for individuals across all ages and varying disease severities.
Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, expressed optimism about the approval, stating, "The European approval of Sephience is a great step in our efforts to bring this safe and highly effective therapy to children and adults affected by PKU worldwide. This broad label supports that potential for Sephience to address all key PKU patient segments and become the new standard of care." This highlights the commitment of PTC to ensuring that its treatments are accessible to all who need them.
Significance of the European Approval
The European approval was based on compelling results from the Phase 3 APHENITY trial which demonstrated significant clinical efficacy. The trial also provided evidence indicating a durable treatment effect, allowing participants to enjoy a more liberalized diet during the long-term extension study. Such findings are crucial as they highlight how Sephience can effectively lower blood phenylalanine (Phe) levels, a critical aspect of managing PKU.
This marketing authorization covers all 27 member states of the European Union, along with Iceland, Norway, and Liechtenstein. PTC Therapeutics intends to kick off the European launch in Germany in the first half of July, marking a strategic initiation of access to essential treatment for PKU patients in Europe.
Ongoing Efforts and Future Aspirations
With the European green light secured, PTC Therapeutics is not resting on its laurels. A New Drug Application (NDA) for sepiapterin remains scheduled for its FDA target action date. The company is also in the process of securing regulatory approvals in other countries, including Japan and Brazil, reflecting their commitment to bringing Sephience to a global audience.
Understanding Sephience™ (sepiapterin) and Its Role
Sephience™ (sepiapterin) is specifically indicated for the treatment of hyperphenylalaninaemia (HPA) in both adults and pediatric patients diagnosed with PKU. Stressing the innovative nature of this treatment, Sephience functions as a natural precursor of the essential enzymatic co-factor BH4, crucial for the proper function of phenylalanine hydroxylase (PAH). This therapy operates as a dual pharmacological chaperone, addressing PAH variants prevalent in PKU that are resistant to standard treatment methods. By enhancing the stability of malfunctioning PAH enzymes and increasing BH4 concentrations intracellularly, Sephience aims to significantly reduce blood Phe levels.
About Phenylketonuria (PKU)
Phe is an essential amino acid necessary for human health, yet for those living with phenylketonuria (PKU), it poses a severe risk. PKU is a hereditary metabolic disorder that results from a genetic defect affecting the enzyme responsible for breaking down Phe. If untreated, elevated levels of Phe can lead to cognitive impairments, developmental delays, and other debilitating health conditions. Early diagnosis through newborn screening can mitigate these risks, yet it is estimated that around 58,000 individuals globally are living with this condition, highlighting the pressing need for effective therapies.
About PTC Therapeutics, Inc.
PTC Therapeutics, Inc. is dedicated to the discovery, development, and commercialization of innovative medicines tailored for children and adults impacted by rare disorders. With a robust pipeline of transformative treatments, PTC is committed to leveraging its global expertise to make a significant difference in the lives of patients. You can learn more about PTC Therapeutics and their offerings by visiting their official website and following them on social media platforms.
Frequently Asked Questions
What is Sephience™ used for?
Sephience™ (sepiapterin) is used to treat phenylketonuria (PKU), helping to lower blood phenylalanine levels in patients.
When will Sephience™ launch in Europe?
The launch of Sephience™ in Europe, starting in Germany, is scheduled for the first half of July.
How does Sephience™ work?
Sephience acts as a pharmacological chaperone, enhancing the activity of defective enzymes to reduce harmful Phe levels.
Is Sephience™ approved in other countries?
Approval processes for Sephience™ are ongoing in several countries, including the United States, Japan, and Brazil.
What is the purpose of the Phase 3 APHENITY trial?
The APHENITY trial evaluated the safety and effectiveness of Sephience™ for individuals with PKU, demonstrating significant positive results.
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