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

Updated: Apr 30, 2023

Novartis' iptacopan demonstrates superior data in PNH-naive patients; the Swiss giant aims at a very broad range of PNH patients with its complement factor B inhibitor

Novartis has announced positive results from a Phase III clinical trial for its investigational oral monotherapy, iptacopan, for patients with paroxysmal nocturnal hemoglobinuria (PNH).

The trial achieved its primary endpoint, with an estimated 92.2% of complement-inhibitor-naive patients achieving an increase of 2 g/dL or more in their hemoglobin levels from baseline without blood transfusions. Secondary endpoints were also met, with the vast majority of patients achieving hemoglobin levels of 12 g/dL or more, independence from blood transfusions, and no cases of clinical breakthrough hemolysis, reduced lactate dehydrogenase levels, and improved patient-reported fatigue.

At 24 weeks, 97.6% of patients achieved independence from a blood transfusion, while 83.55% saw reduced lactate dehydrogenase (LDH) levels.

These results are consistent with the tolerability and safety profiles seen in APPLY-PNH, and they support the potential of iptacopan to be a practice-changing oral medicine for PNH. Novartis aims to treat a broad population of PNH patients with this complement factor B inhibitor.

The Phase III data for C3 glomerulopathy and IgA nephropathy are expected later this year. Iptacopan has received breakthrough designation from the US FDA for treating PNH and orphan designation for C3G and PNH.


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.


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.



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