CHMP Recommends Novartis Fabhalta® for Adult Paroxysmal Nocturnal Hemoglobinuria (PNH)

Novartis Unveils Positive CHMP Endorsement for Fabhalta® in Treating Paroxysmal Nocturnal Hemoglobinuria (PNH)

Novartis has disclosed that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a favorable opinion, recommending the authorization of Fabhalta® (iptacopan) for managing hemolytic anemia in adults diagnosed with paroxysmal nocturnal hemoglobinuria (PNH).

Antonio Risitano, M.D., Ph.D., President of the International PNH Interest Group and Head of the Hematology and Hematopoietic Transplant Unit, commended Fabhalta’s potential impact on PNH patients, emphasizing its capacity to alleviate the burdens associated with the condition. Clinical trials have showcased Fabhalta’s superiority in enhancing hemoglobin levels without necessitating red blood cell transfusions compared to existing anti-C5 therapies, potentially revolutionizing treatment for this chronic blood disorder.

PNH, characterized by hemolysis, bone marrow failure, and thrombosis, poses significant challenges to patients due to its debilitating nature. Current anti-C5 treatments, administered via infusion or injection, often fail to adequately control PNH symptoms, with a substantial proportion of patients experiencing persistent anemia and fatigue despite treatment.

The CHMP’s positive decision stems from comprehensive data from the Phase III APPLY-PNH and APPOINT-PNH studies. APPLY-PNH demonstrated that a significant majority of Fabhalta-treated patients achieved sustained hemoglobin level improvements and avoided transfusions compared to those on anti-C5 therapy. Moreover, Fabhalta effectively controlled intravascular hemolysis and contributed to improved fatigue levels, as evidenced by patient-reported outcomes.

Patrick Horber M.D., President, International at Novartis, underscored the potential significance of Fabhalta as the first oral monotherapy for PNH patients in Europe. If approved by the European Commission, Fabhalta could offer a convenient and effective alternative to current treatment modalities, reducing the burden on patients who often face challenges managing their condition with existing therapies.

With the CHMP’s recommendation, the European Commission is expected to make a final decision on the approval of Fabhalta within the next two months.

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