NICE Recommends Novartis' Fabhalta for Rare Blood Disorder PNH

Novartis’ Fabhalta (iptacopan) has been endorsed by the National Institute for Health and Care Excellence (NICE) for treating paroxysmal nocturnal haemoglobinuria (PNH) in adult patients with haemolytic anaemia. This factor B inhibitor will now be available on the NHS in England and Wales, offering a new treatment option for individuals with this rare blood disorder, which impacts approximately ten to 20 people per million globally.

PNH is characterized by an acquired mutation that makes red blood cells vulnerable to early destruction by the complement system. This condition can lead to a range of issues, including anaemia, thrombosis, and fatigue, which significantly affect patients' quality of life.

With Fabhalta, patients now have access to the first oral monotherapy for PNH, moving away from the reliance on infusions that have been the traditional treatment approach. The NICE approval follows last month’s approval of the therapy by the Medicines and Healthcare products Regulatory Agency, supported by data from the late-stage APPLY-PNH trial. This trial focused on adults with PNH who still had anaemia despite previous anti-C5 treatment. Findings indicated that patients who switched to Fabhalta showed superior improvements in haemoglobin levels compared to those who continued with anti-C5 therapy.

Furthermore, the decision by NICE was also backed by results from the phase 3 APPOINT-PNH study, which involved complement inhibitor-naïve patients. Austin Kulasekararaj, a consultant haematologist at King’s College Hospital, highlighted that data from both the APPLY-PNH and APPOINT-PNH trials demonstrated Fabhalta’s efficacy in controlling haemolysis and boosting haemoglobin levels in PNH patients. He noted that the availability of this oral treatment marks a significant advance for the national PNH service.

In December, Fabhalta received approval from the US Food and Drug Administration (FDA) for the treatment of adults with PNH. The therapy has also been recommended by the European Medicines Agency’s human medicines committee for the same indication.

Moreover, last month, the FDA granted accelerated approval for Fabhalta to reduce excess protein in the urine of patients with immunoglobulin A nephropathy, a rare kidney disease. The drug is also being developed for other rare diseases, including C3 glomerulopathy, immune complex membranoproliferative glomerulonephritis, atypical haemolytic uraemic syndrome, and lupus nephritis.

This series of approvals and recommendations highlights the broad potential of Fabhalta in treating a variety of rare diseases, providing new hope for patients with these challenging conditions.

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