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Orphan Drug Designation Granted for CSL Behring’s Investigational Plasma-Derived Hemopexin Therapy for Sickle Cell Disease
Global biotherapeutics leader CSL Behring announced today that its investigational, plasma-derived hemopexin therapy (CSL889) received orphan drug designation from both the European Commission and the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for the treatment of sickle cell disease (SCD). These designations grant special status to drugs and biological products intended to treat a rare disease, affecting less than 200,000 patients in the US or affecting not more than five in 10,000 people in the European Union.
CSL889 is a form of plasma-derived hemopexin, an important, naturally occurring protein produced in the body whose levels are decreased in patients with SCD. Low levels of hemopexin have been associated with increased symptoms in SCD, particularly acute vaso-occlusive crises (VOC).
education & researchA Five Fold Decrease in Admissions for Uncomplicated Vaso-Occlusive Crisis and Other Benefits from Care in Infusion ...Patients with sickle cell disease (SCD) ...
news & eventsNew Guidelines for Managing Sickle Cell Disease Presented at ACP Internal Medicine MeetingNew guidelines for therapeutic strategie...
education & researchIdentifying central sensitization in adults with sickle cell disease: Differences in clinical features and psychobeh...Central sensitization (CS) has been iden...
news & eventsGBT expands sickle cell disease pipeline with worldwide licensing agreement for inclacumab for the treatment of vaso...Global Blood Therapeutics, Inc...
videos & visualsVaso occlusive Crisis Pain Assessment & Managementhttps://www.youtube.com/watch?time_conti...
news & eventsWhen Cracking Down On Opioids Means Tougher Access For Sickle Cell PatientsMany people with sickle cell disease wil...
news & eventsAPS Scientific Meeting 2019The American Pain Society will hold its ...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.