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Pint Pharma's EMPAVELI® (pegcetacoplan) Receives Regulatory Approval as the First Targeted Therapy for Paroxysmal Nocturnal Hemoglobinuria (PNH) in Adults

Thursday, July 27, 2023

OrphanDC and Pint Pharma Secure ANVISA Approval for EMPAVELI® (pegcetacoplan), a Groundbreaking Targeted Therapy for Paroxysmal Nocturnal Hemoglobinuria (PNH) in Adults. Empaveli's Novel Mechanism Involves Binding to the Complement Protein C3, Revolutionizing PNH Treatment.

Pint Pharma and OrphanDC have achieved a significant milestone with ANVISA's approval of Empaveli® (pegcetacoplan) in Brazil. This drug represents a breakthrough in treating Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare, chronic, and life-threatening blood disorder characterized by the destruction of red blood cells due to uncontrolled complement activation.

PNH patients often experience low hemoglobin levels, leading to frequent transfusions and severe fatigue caused by anemia. Despite improvements with C5 inhibitor therapies, many PNH patients (around 72%, according to a retrospective cross-sectional study) still suffer from anemia.

Empaveli's approval is based on the positive results of the phase 3 PEGASUS study, which demonstrated its efficacy and safety compared to eculizumab over 16 weeks in adults with PNH. The therapy targets complement protein C3, making it the first C3-targeted therapy in Brazil, offering hope for enhanced quality of life for PNH patients.

David Muñoz, CEO, and President of Pint Pharma, expressed that Empaveli's approval is a significant milestone for people living with PNH in Brazil, as the symptoms of PNH can significantly impact patients' quality of life.

Valnei Canutti, Hematologist, and Head of Scientific Affairs at Pint Pharma, highlighted that Empaveli's approval represents a therapeutic innovation for PNH patients, based on the positive results from the PEGASUS study.

Daniel Zequeto, Founder, and CEO of OrphanDC emphasized that Empaveli's approval marks an innovative breakthrough in PNH treatment, as it represents the first registration of a new class of drugs targeting complement C3, potentially leading to a remarkable improvement in patients' quality of life.

Flavio Santoro, General Manager of Pint Pharma in Brazil, expressed that Empaveli's approval is not just a victory for the company but more importantly, a victory for patients who will now have access to this innovative therapy, which has the potential to significantly improve the lives of those affected by this devastating disease.

Empaveli, known as Aspeveli in the European Union and Empaveli in the United States, is designed to regulate excessive activation of the complement cascade, an important part of the body's immune system implicated in the onset and progression of several serious diseases. In addition to PNH, Empaveli is under investigation for several other rare diseases in the fields of hematology, nephrology, and neurology.

Empaveli will be exclusively marketed and distributed in Brazil by Pint Pharma, following its registration by OrphanDC G Importação e Distribuição de Produtos Farmacêuticos.

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