First-in-class, oral, targeted factor B inhibitor iptacopan substantially reduced both intra- and extravascular hemolysis when given as monotherapy in a Phase II study of anti-C5 naïve paroxysmal ...

“In a phase 2 study, iptacopan controlled intra- and extravascular hemolysis in 10 patients with a suboptimal response to eculizumab, leading to transfusion independence and [improved] quality ...

Iptacopan showed superiority (P<0.0001) for reduction in absolute reticulocyte count from baseline (a marker of control for extravascular hemolysis) and in annualized rate of breakthrough ...

Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected as a pharmacy partner by Novartis for Fabhalta® (iptacopan) as the fi ...

Ad hoc announcement pursuant to Art. 53 LR Approval based on APPLY-PNH trial in adults with PNH and anemia despite prior anti-C5 treatment, and supported by the APPOINT-PNH study in complement ...

To manage patients’ hemolysis, 68% (n = 13) underwent a dose increase of pegcetacoplan from twice weekly to 1080 mg every 3 days. This approach reportedly benefited 6 patients’ hemolytic outcomes.

The approval was based on data from the phase 3 ALPHA trial, which evaluated danicopan as add-on therapy to ravulizumab or eculizumab in adults with PNH and clinically significant EVH.

In clinical trials, treatment with Fabhalta increased hemoglobin levels in most patients, and in APPLY-PNH, nearly all patients treated with Fabhalta did not receive blood transfusions.

FDA OKs Danicopan Add-On for Extravascular Hemolysis in Adults With PNH. M. Alexander Otto, PA, MMS. April 02, 2024 . 0. 0.

Principal support for the approval came from the randomized phase III ALPHA trial, which evaluated standard treatment with or without danicopan in 73 patients with PNH and significant ...

The first-in-class oral factor D inhibitor danicopan (Voydeya) has been approved for the management of extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH).

FDA Approves Alexion’s Voydeya as Add-on Treatment for Adults with Extravascular Hemolysis. April 1, 2024. By Don Tracy, Associate Editor. News. Article. Treatment approved in combination with ...