DISCLAIMER
The information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.compose your message
message sent
email sent successfully
Trusted Resources: News & Events
Latest announcements and gatherings
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).
Related Content
-
education & researchAcute Vaso-Occlusive Episode in Sickle Cell AnemiaAcute Vaso-Occlusive Episode in Sickle C...
-
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 & eventsCrizanlizumab designated FDA breakthrough therapy for potential in vaso-occlusive crisis preventionCrizanlizumab (SEG101), Novartis‘ inve...
-
Community CenterLiving With Sickle Cell Disease Is a Constant Battle With Pain, According to one Woman Who Has It“You don’t look sick.” That’s ...
-
news & eventsResearch Team Recognized for Organ-on-a-Chip DesignSCD is a group of genetic disorders that...
-
videos & visualsPatient Perspective: The Journey of Pain in Sickle Cell Diseasehttps://www.youtube.com/watch?v=F4raFO0e...
-
news & eventsGut Microbiome Translates Stress Into Sickle Cell CrisesA new study shows how chronic psychologi...
send a message
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Support for this site is provided by
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.