Novartis Fabhalta (iptacopan) receives positive CHMP opinion as first oral monotherapy for adult patients with paroxysmal nocturnal hemoglobinuria (PNH)

• Positive CHMP opinion based on robust Phase III data, including APPLY-PNH, demonstrating superior hemoglobin improvement in the absence of transfusions with Fabhalta compared to anti-C5 therapy^1-5
  
• If approved, Fabhalta will be the first oral monotherapy available in Europe for the treatment of PNH, a chronic and rare blood disorder, in both complement inhibitor-treated and -naive PNH patients with hemolytic anemia¹
• Despite anti-C5 therapy, a large proportion of patients remain anemic, fatigued and dependent on blood transfusions^6,7 
  
• Late-stage Fabhalta development program is ongoing in multiple complement-mediated diseases
  

Basel, March 22, 2024 – Novartis announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion and recommended granting a marketing authorization for Fabhalta (iptacopan) for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia¹. 

“With a robust body of evidence and a demonstrated safety profile, Fabhalta could be practice-changing for patients, helping relieve burdens experienced by people living with PNH,” said Antonio Risitano, M.D., Ph.D., President of the International PNH Interest Group and Head of the Hematology and Hematopoietic Transplant Unit, Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria at the AORN San Giuseppe Moscati, Avellino, Italy. “In clinical studies, oral iptacopan demonstrated superior hemoglobin improvement without the need for red blood cell transfusions compared to anti-C5 therapies, leading to normalization of hemoglobin in the majority of patients—a potentially groundbreaking benefit for those living with this chronic blood disorder.” 

PNH is a rare and debilitating chronic blood disorder that occurs when blood cells which are intrinsically susceptible to a part of the immune system called the complement system expand over normal blood cells due to a permissive immune environment^8-11. PNH is characterized by hemolysis, bone marrow failure, and thrombosis in varying combinations and levels of severity^6,7,12. Current anti-C5 treatments are administered via infusion or subcutaneous injection and may leave PNH symptoms uncontrolled. Up to 50% of patients on anti-C5 treatment may have persistent anemia with 23-39% remaining dependent on blood transfusions, and the majority (75-89%) of patients on anti-C5 treatment remain fatigued^7,13-17.