Novartis has achieved a milestone with the FDA approval of iptacopan, now branded as Fabhalta, for treating paroxysmal nocturnal hemoglobinuria (PNH). This makes it the inaugural oral monotherapy greenlit by the FDA for this rare blood disorder. Fabhalta offers a contrasting approach to existing PNH therapies like Soliris (eculizumab) and Ultomiris (ravulizumab) by AstraZeneca/Alexion, as well as Apellis’ Empaveli (pegcetacoplan), which necessitate infusion or injection. Its approval encompasses both previously treated and treatment-naïve patients, establishing a broad label for its use.
Novartis foresees a substantial impact from Fabhalta’s approval, suggesting it might redefine patients’ expectations concerning PNH. Analysts project a peak annual revenue potential of $3.6 billion if iptacopan is approved for its various targeted indications, including atypical hemolytic uraemic syndrome (aHUS) and rare kidney diseases like C3 glomerulopathy (C3G) and idiopathic membranous nephropathy (IMN). Novartis itself anticipates a revenue potential of $3 billion or more from this drug.
Addressing the Treatment Gap in Paroxysmal Nocturnal Hemoglobinuria
PNH is characterized by the body’s complement system destroying red blood cells, causing anemia, fatigue, and, in severe cases, necessitating frequent blood transfusions. Approximately 10-20 people per million globally live with PNH, commonly diagnosed in individuals between 30-40 years old.
Despite existing complement therapies, a significant treatment gap persists in PNH, with many patients experiencing ongoing anemia and reliance on blood transfusions. Between 20% and 50% of PNH patients treated with drugs like Soliris can’t achieve symptom control and require blood transfusions, indicating an urgent need for alternative treatment options.
Fabhalta Paving the Way for Transformative PNH Treatment
The FDA clearance for Fabhalta is founded on the outcomes of the APPLY-PNH and APPOINT-PNH trials. These trials demonstrated clinically meaningful improvements in hemoglobin levels, particularly in patients with persistent anemia despite previous anti-C5 antibody treatments and those untreated with C5 inhibitors.
In these trials, Fabhalta showcased notable efficacy by significantly increasing hemoglobin levels, surpassing the performance of C5 drugs. Notably, nearly all patients administered Fabhalta in the APPLY-PNH trial did not require blood transfusions.
Dr. Vinod Pullarkat from the City of Hope in California highlighted the potential impact of effective oral treatment, stressing its capacity to change practices among physicians and alleviate the burdens faced by PNH patients. This pivotal approval holds promise for transforming the landscape of PNH treatment by offering an effective, orally administered option with a demonstrated safety profile.
Resource: Pharmaphorum, December 06, 2023
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