Google
Trusted Resources: News & Events
Latest announcements and gatherings
FDA Approves Novel Treatment to Target Abnormality in Sickle Cell Disease
Today, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older.
“Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder,” said Acting FDA Commissioner Adm. Brett P. Giroir, M.D. “Our scientific investments have brought us to a point where we have many more tools available in the battle against sickle cell disease, which presents daily challenges for those living with it. We remain committed to raising the profile of this disease as a public health priority and to approving new therapies that are proven to be safe and effective. Together with improved provider education, patient empowerment, and improved care delivery systems, these newly approved drugs have the potential to immediately impact people living with SCD.”
Sickle cell disease is a lifelong, inherited blood disorder in which red blood cells are abnormally shaped (in a crescent, or “sickle” shape), which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. It is also characterized by severe and chronic inflammation that worsens vaso-occlusive crises during which patients experience episodes of extreme pain and organ damage. Nonclinical studies have demonstrated that Oxbryta inhibits red blood cell sickling, improves red blood cell deformability (ability of a red blood cell to change shape) and improves the blood’s ability to flow.
+myBinderRelated Content
-
Bryan B. Bushman, PhDBryan Bushman, Ph.D., is a licensed clin...
-
Lakshmanan Krishnamurti, MDDr. Krishnamurti graduated from the Arme...
-
Sickle Cell Disease Association of America – Mobile Chapter, Inc.The Sickle Cell Disease Association of A...
-
Predictive factors of daily opioid use and quality of life in adults with sickle cell diseaseObjectives: In adults with sickle cell ...
-
Development of a Pharmacokinetic‐Guided Dose Individualization Strategy for Hydroxyurea Treatment in Children With...Aims: Hydroxyurea has emerged as the p...
-
Alzheimer’s Treatment Memantine Shows Promise in Treating Sickle Cell DiseaseMemantine, a standard treatment for Alzh...
-
Developing Hydroxyurea, the First FDA-Approved Therapy for Sickle Cell Diseasehttps://www.youtube.com/watch?v=0kIz5ZKy...
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.
Powered by
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.