PTC Therapeutics' Sephience™ Receives FDA Approval for PKU

PTC Therapeutics Achieves FDA Milestone with Sephience™ Approval

PTC Therapeutics, Inc. (NASDAQ: PTCT) is thrilled to announce that the U.S. Food and Drug Administration (FDA) has granted approval for SEPHIENCE™ (sepiapterin) as a treatment for individuals of all ages living with phenylketonuria (PKU). This significant decision allows the use of SEPHIENCE for both children and adults beginning from one month of age. The comprehensive labeling also encompasses various disease subtypes, offering hope and a potential change in care for many affected by PKU.

"This is an important milestone for those dealing with PKU," conveyed Matthew B. Klein, M.D., the Chief Executive Officer of PTC Therapeutics. He emphasized that the extensive labeling demonstrates Sephience's potential in addressing the significant unmet needs of PKU patients. With robust clinical data and their expertise in rare disease treatments, PTC is poised to introduce Sephience effectively, aiming to enhance the quality of life for families affected by this condition.

The Journey to FDA Approval

The FDA's decision was driven by substantial evidence arising from the Phase 3 APHENITY trial along with notable results from a long-term extension study. These studies confirmed SEPHIENCE's efficacy and safety, establishing it as a vital option for managing PKU and hyperphenylalaninemia (HPA) in patients who are responsive to sepiapterin.

Catherine Warren, the Executive Director of the National PKU Alliance, shared her excitement regarding this approval, highlighting its importance for the PKU community. She mentioned how the new treatment option brings renewed hope and how its positive impact might significantly enhance the care provided to individuals of all ages and subtypes of PKU.

Looking Beyond the U.S. Market

SEPHIENCE recently received marketing authorization from the European Commission, signaling PTC's commitment to making this important medication available globally. PTC is also in discussions regarding approval applications in several countries, including Japan and Brazil. This international focus reflects the company's dedication to expanding access to innovative therapies across borders.

Details on SEPHIENCE™

SEPHIENCE™ serves as a crucial treatment aimed at significantly reducing blood phenylalanine (Phe) levels in patients diagnosed with PKU. By acting as a natural precursor to the enzymatic co-factor BH4, sepiapterin offers a new avenue for patients whose condition is compatible with this treatment. In conjunction with a phenylalanine-restricted diet, SEPHIENCE™ is expected to provide a much-needed alternative for effective PKU management.

Conference Call and Interaction

In light of this announcement, PTC plans to hold a conference call to delve deeper into the implications of this FDA approval. Scheduled for a future date, details will be shared to ensure interested individuals can join in. Participants are advised to connect earlier to avoid delays.

Indication and Safety for SEPHIENCE™

The application of SEPHIENCE is strictly for treating hyperphenylalaninemia in patients aged one month and older with sepiapterin-responsive PKU. This therapy requires careful management by qualified healthcare providers to monitor the safety and dietary patterns necessary for successful treatment.

Warnings and Precautions for Patients

As with any new medication, careful consideration is warranted. SEPHIENCE may increase the risk of bleeding in some patients, and monitoring is crucial to ensure the safety of individuals, especially pediatric patients who may experience fluctuating blood Phe levels.

PTC Therapeutics is steadfast in its mission to develop differentiated medicines focused on individuals with rare disorders. They strive to bring transformative therapies that not only enhance patient care but also improve the overall quality of life for families navigating the challenges of PKU.

Frequently Asked Questions

What is SEPHIENCE™ used for?

SEPHIENCE™ is indicated for the treatment of hyperphenylalaninemia in patients with phenylketonuria (PKU).

Who can receive treatment with SEPHIENCE™?

This treatment is suitable for both children and adults from the age of one month and older who are diagnosed with PKU.

Where has SEPHIENCE™ received approval?

SEPHIENCE™ has been approved in the United States and recently received marketing authorization in the European Union.

What potential risks are associated with SEPHIENCE™?

Patients may experience an increase in bleeding risks. Continuous monitoring is necessary to manage this potential side effect.

What is the commitment of PTC Therapeutics regarding SEPHIENCE™?

PTC Therapeutics is committed to providing access to innovative treatments and is focused on ongoing research and marketing efforts globally to help patients manage PKU effectively.

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