Novartis’ Fabhalta (iptacopan) has been recommended by the European Medicines Committee to treat paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia.
If approved, Fabhalta would be the first oral monotherapy available in Europe to treat patients with the rare blood disorder affecting approximately ten to 20 people per million worldwide.
PNH patients have an acquired mutation that causes them to produce red blood cells susceptible to premature destruction by the complement system, potentially leading to anaemia, thrombosis, fatigue and other symptoms that can impact quality of life.
Current anti-C5 treatments are administered via infusion or subcutaneous injection and may leave PNH symptoms uncontrolled, Novartis outlined, with up to 50% of those on anti-C5 treatment experiencing persistent anaemia and up to 39% remaining dependent on blood transfusions.
Fabhalta is a factor B inhibitor of the immune system’s complement pathway, which drives complement-mediated haemolysis in PNH.
The decision from the Committee for Medicinal Products for Human Use (CHMP) on the therapy was supported by data from the late-stage APPLY-PNH trial in adults with PNH and residual anaemia despite prior anti-C5 treatment, which showed that patients who switched to Fabhalta experienced superior increases in haemoglobin levels compared to those who continued on anti-C5 treatment.
Results from the phase 3 APPOINT-PNH study in complement inhibitor-naïve patients also supported the CHMP’s recommendation.
Patrick Horber, president, international, Novartis, 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.’’
Fabhalta was approved by the US Food and Drug Administration as the first oral monotherapy for adults with PNH in December, with Victor Bultó, president US, Novartis, describing the authorisation as an “extraordinary moment” for the PNH community.
The therapy is also currently in development for a range of other complement-mediated diseases, including immunoglobulin A nephropathy, C3 glomerulopathy, immune complex membranoproliferative glomerulonephritis and atypical haemolytic uraemic syndrome.
