Novartis has reported positive topline outcomes from the multicentre Phase IIIB APPULSE-PNH trial of oral Fabhalta (iptacopan) for treating adults with paroxysmal nocturnal haemoglobinuria (PNH).
These subjects transitioned from anti-C5 therapies with a baseline haemoglobin (Hb) level of at least 10g/dL after treatment with eculizumab or ravulizumab.
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The open-label, single-arm trial involved 52 subjects. It is designed to assess the efficacy and safety of 200mg Fabhalta in adults who were switched from anti-C5 therapies, eculizumab or ravulizumab.
To qualify, participants had to be on a stable regimen of anti-C5 therapies for at least six months, with no red blood cell transfusions during this period.
The trial’s primary goal is change from baseline haemoglobin (Hb) levels post the 24-week treatment period.
Subjects exhibited an improvement in average Hb levels compared to baseline. The therapy’s safety profile was consistent with prior data.
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By GlobalDataNovartis Cardiovascular, Renal and Metabolism Development Unit global head David Soergel said: “Across multiple clinical trials, Fabhalta has consistently shown clinically meaningful benefits for patients with PNH, and the APPULSE-PNH trial is a compelling addition to this body of evidence.
“These data reinforce our confidence in Fabhalta, the first and only oral monotherapy currently available for the treatment of adults with PNH, to provide meaningful haemoglobin improvement, regardless of previous treatment experience.”
PNH is a blood disorder where mutated hematopoietic stem cells produce red blood cells that are prone to destruction by the complement system.
It affects approximately 10-20 individuals per million globally and is often diagnosed in individuals in the 30-40 age group.
The US Food and Drug Administration (FDA) recently granted accelerated approval to the therapy for decreasing proteinuria in the adult population with primary immunoglobulin A nephropathy (IgAN), and its development continues in various complement-mediated diseases in August this year.
In October this year, the company shared positive outcomes from the Phase III APPEAR-C3G study of Fabhalta, along with supportive care, for subjects with C3 glomerulopathy (C3G) over a 12-month period.
