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Paroxysmal nocturnal hemoglobinuria (PNH) News | Blogs | Treatment | Updates | iPharmaCenter

Paroxysmal nocturnal hemoglobinuria is a rare, acquired blood disorder. It can be fatal and is characterized by the destruction of blood cells and impaired bone marrow function.

Soliris, the most widely used therapy for PNH, binds to the proteins involved in destroying the blood cells. Ultomiris is also approved for treating PNH.

Bone marrow transplant is recommended in patients in whom Soliris and Ultomiris don't work.


Novartis oral therapy showed superiority over AstraZeneca's blockbuster Soliris and Ultomiris.

Novartis announced that its investigational oral therapy, iptacopan, showed meaningful superiority over anti-C5 therapeutics treatments, Soliris (eculizumab), and Ultomiris (ravulizumab) in patients with paroxysmal nocturnal hemoglobinuria (PNH) previously treated with anti-C5-treatments.

Novartis announced the preliminary results of Phase 3 APPLY-PNH active-controlled trial, aimed at demonstrating the efficacy of iptacopan versus Soliris and Ultomiris. The co-primary endpoints were the increase in hemoglobin levels by ≥ 2 g/dL and the maintenance of sustained hemoglobin levels of ≥ 12 g/dL at day 168. Novartis announced that iptacopan showed significant improvement in both the primary endpoints compared to anti-C5 therapies.

Iptacopan is in development for complement-mediated kidney diseases (CMKDs) C3 glomerulopathy, IgA nephropathy, and atypical hemolytic uremic syndrome. Novartis is aiming for the regulatory submissions of iptacopan starting from 2023.

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