Novartis’ Fabhalta (iptacopan) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia.
The factor B inhibitor will be available immediately on the NHS in England and Wales to treat patients with the rare blood disorder that affects 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.
Fabhalta is now the first oral monotherapy for this patient population, who until now have relied on infusions as the standard of care.
NICE’s final guidance follows the Medicines and Healthcare products Regulatory Agency’s approval of the therapy last month and 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 decision.
Austin Kulasekararaj, consultant haematologist at King’s College Hospital, said: “Data from the APPLY-PNH and APPOINT-PNH trials showed that [Fabhalta] can be effective in controlling haemolysis and improving haemoglobin levels in patients with PNH. [This] news means our… national PNH service now has access to the first oral treatment option for this rare disease.”
Fabhalta was approved by the US Food and Drug Administration (FDA) for adults with PNH in December, and has since been recommended by the European Medicines Agency’s human medicines committee for the same indication.
The therapy was granted accelerated approval by the FDA last month to reduce excess protein in the urine of patients with the rare kidney disease immunoglobulin A nephropathy.
It is also currently in development for a range of rare diseases, including C3 glomerulopathy, immune complex membranoproliferative glomerulonephritis, atypical haemolytic uraemic syndrome and lupus nephritis.
