Novartis: Fabhalta Gets Positive CHMP Opinion To Treat Paroxysmal Nocturnal Hemoglobinuria

(RTTNews) - Swiss drug major Novartis AG (NVS) announced Friday that Fabhalta (iptacopan) has received a positive opinion from the Committee for Medicinal Products for Human Use or CHMP of the European Medicines Agency for the treatment of adults with paroxysmal nocturnal hemoglobinuria or PNH who have hemolytic anemia.

The CHMP has recommended granting a marketing authorization for the drug. Following the recommendation, the European Commission will take a final decision within approximately two months.

If approved, Fabhalta would be the first oral monotherapy for adult patients in Europe with PNH, a rare and debilitating chronic blood disorder.

PNH 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.

The company noted that PNH is characterized by hemolysis, bone marrow failure, and thrombosis in varying combinations and levels of severity.

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. It is also based on Phase III APPOINT-PNH study in complement-inhibitor naïve patients.

In clinical studies, oral iptacopan demonstrated superior hemoglobin improvement without the need for red blood cell transfusions compared to anti-C5 therapies. This lead to normalization of hemoglobin in the majority of patients.

In both studies, Fabhalta was shown to control the destruction of red blood cells within the blood vessels, known as intravascular hemolysis.

The company said the safety profile of iptacopan was consistent across both APPLY-PNH and APPOINT-PNH studies.

Patrick Horber President, International, Novartis, Basel, said, "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."

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