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  • Novartis Fabhalta gets...

Novartis Fabhalta gets positive CHMP opinion as first oral monotherapy for adult patients with paroxysmal nocturnal hemoglobinuria

Written By : Ruchika Sharma |Medically Reviewed By : Dr. Kamal Kant Kohli Published On 2024-03-24T14:30:13+05:30  |  Updated On 24 March 2024 2:30 PM IST
Novartis to acquire kidney disease drug maker Regulus Therapeutics for up to 1.7 billion
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Basel: Novartis has announced 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 anemia.

“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. PNH is characterized by hemolysis, bone marrow failure, and thrombosis in varying combinations and levels of severity. 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.

The positive CHMP decision is based on robust data from the Phase III APPLY-PNH study in patients with residual anemia despite prior anti-C5 treatment who switched to Fabhalta vs. patients who stayed on anti-C5 treatment, and the Phase III APPOINT-PNH study in complement-inhibitor naïve patients. In APPLY-PNH, at 24 weeks, 82.3% of anti-C5-experienced Fabhalta patients achieved a sustained increase of Hb levels ≥2 g/dL from baseline in the absence of transfusions vs. 2.0% for anti-C5 (difference of 80.2%, P<0.0001); in APPOINT-PNH, 92.2% of complement inhibitor-naïve patients using Fabhalta achieved this outcome. Similarly, in APPLY-PNH, results highlighted a transfusion avoidance rate of 94.8% for anti-C5-experienced Fabhalta patients vs. 25.9% for those on anti-C5 (difference of 68.9%, P<0.0001). In both studies, Fabhalta was also shown to control the destruction of red blood cells (RBCs) within the blood vessels, known as intravascular hemolysis (IVH), with mean lactate dehydrogenase (LDH) levels maintained at <1.5 x upper limit of normal. In APPLY-PNH, patients reported improvements in fatigue as measured by Functional Assessment of Chronic Illness Therapy – Fatigue [FACIT-F] scores. The safety profile of iptacopan was consistent across both APPLY-PNH and APPOINT-PNH studies.

“If approved by the European Commission, Fabhalta would be the first oral monotherapy available to PNH patients in Europe,” said Patrick Horber M.D., President, International, Novartis, Basel. “With current standard of care, PNH symptoms are often uncontrolled, while patients endure regular and time-consuming infusions. This oral therapy could provide a much-needed alternative to support many people living with PNH who often have to structure their lives around managing their condition.’’

Following the CHMP’s recommendation to approve Fabhalta in adult patients with PNH who have hemolytic anemia, the European Commission (EC) will take a final decision within approximately two months.

Read also: CDSCO Panel Approves Novartis Protocol Amendment Proposal For Multiple Sclerosis Drug Ofatumumab

novartisnovartis newsfabhaltaiptacopanparoxysmal nocturnal hemoglobinuriahemolytic anemia
Ruchika Sharma
Ruchika Sharma

    Ruchika Sharma joined Medical Dialogue as an Correspondent for the Business Section in 2019. She covers all the updates in the Pharmaceutical field, Policy, Insurance, Business Healthcare, Medical News, Health News, Pharma News, Healthcare and Investment. She has completed her B.Com from Delhi University and then pursued postgraduation in M.Com. She can be contacted at editorial@medicaldialogues.in Contact no. 011-43720751

    Dr. Kamal Kant Kohli
    Dr. Kamal Kant Kohli

    Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as a Chief Editor of Medical News. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Email: drkohli@medicaldialogues.in. Contact no. 011-43720751

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