Novartis Receives FDA Approval for Fabhalta (iptacopan), the First Oral Monotherapy for Paroxysmal Nocturnal Hemoglobinuria

Dec 06, 2023 09:03 CST Updated 09:03

Novartis

Drug Development and Manufacturing

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Novartis today announced,The US FDA Approves Fabhalta (iptacopan) as the First Oral Monotherapy for Treating Adult Paroxysmal Nocturnal Hemoglobinuria (PNH)Fabhalta is a complement factor B inhibitor that acts on the alternative complement pathway of the immune system, providing comprehensive control over the destruction of red blood cells (RBCs) both inside and outside blood vessels. Clinical trials have shown that Fabhalta effectively increases hemoglobin levels in most patients, and in the Phase 3 clinical trial APPLY-PNH, nearly all patients using Fabhalta did not require transfusions. This drug was listed by the industry media Evaluate as10 Potential Blockbuster Therapies Worth WatchingOne of.

The FDA approval is based on the Phase 3 clinical trial APPLY-PNH. This trial included patients with hemoglobin <10 g/dL who had previously been treated with anti-C5 therapy but still had residual anemia. The results showed that Fabhalta was superior to continuing anti-C5 therapy in improving hemoglobin levels and avoiding transfusions. The approval was also supported by the Phase 3 clinical trial APPOINT-PNH, which focused on patients who had not received complement inhibitor treatment. The primary findings of the 24-week APPLY-PNH and APPOINT-PNH trials include:

82.3% of patients previously treated with anti-C5 therapy experienced a sustained increase in hemoglobin levels of ≥2 g/dL without the need for transfusions after receiving Fabhalta., the value for patients who continued to receive anti-C5 therapy was 0% (P<0.0001). Among patients who had not received complement inhibitor therapy,77.5%Patients treated with Fabhalta achieved this outcome.
Without blood transfusion,67.7% of patients treated with anti-C5 therapy maintained hemoglobin levels ≥12 g/dL, the value for patients who continued to receive anti-C5 therapy was 0% (P<0.0001).
For patients who have received anti-C5 therapy,The transfusion avoidance rate after receiving Fabhalta treatment is 95.2%., while the value for patients in the anti-C5 therapy group was 45.7% (P<0.0001).

▲Introduction to Iptacopan (Image Source: Novartis official website)

In the APPLY-PNH trial, the most frequently reported adverse reactions (≥10%) for Fabhalta compared to anti-C5 therapy included: headache (19% vs 3%), nasopharyngitis (16% vs 17%), diarrhea (15% vs 6%), abdominal pain (15% vs 3%), bacterial infections (11% vs 11%), nausea (10% vs 3%), and viral infections (10% vs 31%). In the APPOINT-PNH trial, the most frequently reported adverse reactions (≥10%) included headache (28%), viral infections (18%), nasopharyngitis (15%), and rash (10%). In APPLY-PNH, two patients (3%) with PNH treated with Fabhalta reported serious adverse reactions, including pyelonephritis, urinary tract infection, and COVID-19. In APPOINT-PNH, two patients (5%) with PNH treated with Fabhalta reported serious adverse reactions, including COVID-19 and bacterial pneumonia.

Fabhalta is one of Novartis' key research and development projects, and in addition to being used for the treatment of PNH,It is used in the treatment of IgA nephropathy.Phase 3 Clinical TrialIn China, it has also reached the primary endpoint and is expected to file for accelerated approval with regulatory authorities in 2024.

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References:

[1] Novartis receives FDA approval for Fabhalta® (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH. Retrieved December 5, 2023, from https://www.globenewswire.com/news-release/2023/12/06/2791405/0/en/Novartis-receives-FDA-approval-for-Fabhalta-iptacopan-offering-superior-hemoglobin-improvement-in-the-absence-of-transfusions-as-the-first-oral-monotherapy-for-adults-with-PNH.html

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