Παρασκευή 28 Δεκεμβρίου 2018

Ravulizumab for treatment of paroxysmal nocturnal hemoglobinuria

FDA approves ravulizumab for treatment of paroxysmal nocturnal hemoglobinuria
https://www.mdedge.com/internalmedicine/article/191740/anemia/fda-approves-ravulizumab-treatment-paroxysmal-nocturnal
Lucas Franki, Internal Medicine News

Dec 21st, 2018 - The Food and Drug Administration has approved ravulizumab(Ultomiris) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). Wikimedia Commons/FitzColinGerald/Creative Commons License "The approval of Ultomiris will change the way that patients with PNH are treated.

Ravulizumab (ALXN1210) in patients with paroxysmal nocturnal hemoglobinuria: res... Preview
https://www.ncbi.nlm.nih.gov/pubmed/30171081
Blood Advances; Röth A, Rottinghaus ST et. al.

Sep 2nd, 2018 - Ravulizumab (ALXN1210), a humanized monoclonal antibody to complement component C5, was engineered from eculizumab to have a substantially longer terminal half-life, permitting longer dosing intervals for paroxysmal nocturnal hemoglobinuria (PNH) treatment. Two phase 1b/2 multicenter open-label studies evaluated efficacy and safety of multiple doses and regimens of ravulizumab in ...

Drug is convenient alternative for PNH, doc says
https://www.mdedge.com/content/drug-convenient-alternative-pnh-doc-says
Jen Smith

Jun 22nd, 2018 - Photo from EHA Session at the 23rd Congress of the European Hematology Association (EHA) STOCKHOLM—Results of a phase 3 study suggest the long-acting C5 complement inhibitor ravulizumab produces similar results as eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH). Treatment with ravulizumab every 8 weeks proved noninferior to treatment with eculizumab every 2 w.

New Drug for PHN Taken Only Once Every 8 Weeks
https://www.medscape.com/viewarticle/898793

Jul 2nd, 2018 - STOCKHOLM, Sweden — A new drug under development for the treatment of paroxysmal nocturnal hemoglobinuria (PNH),  ravulizumab (ALXN1210, Alexion Pharmaceuticals), has shown noninferiority compared with eculizumab (Soliris, Alexion Pharmaceuticals), currently the only therapy approved for the syndrome. But it achieved similar outcomes while being administered by intravenous (IV) infusion ...

FDA OKs New Options for Two Rare Blood Diseases
https://www.medpagetoday.com/hematologyoncology/hematology/77084

Dec 21st, 2018 - WASHINGTON -- The FDA announced new approvals for two rare and life-threatening blood diseases on Friday, as blastic plasmacytoid dendritic cell neoplasm (BPDCN) gets its first approved agent and paroxysmal nocturnal hemoglobinuria (PNH) gets a new drug that drastically cuts the frequency of treatments. BPDCN Gets First Approved Drug The FDA approved the infusion therapy tagraxofusp (Elzonris) ...

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