Google
Trusted Resources: News & Events
Latest announcements and gatherings
FDA Advisory Committee Recommends Approval of Endari™ from Emmaus Life Sciences for the Treatment of Sickle Cell Disease
Emmaus Life Sciences Inc. announced today that the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) voted 10 to 3 that the overall Benefit-Risk profile of EndariTM for the treatment of sickle cell disease (SCD) is favorable. The FDA has set a PDUFA target action date for July 7, 2017.
If approved, Endari would be the first FDA-approved treatment for pediatric patients with SCD, and the first new treatment in almost 20 years for adult patients. The therapy is an orally-administered pharmaceutical grade L-glutamine.
The FDA is not bound by the Committee’s recommendation, but takes its advice into consideration when reviewing New Drug Applications. Endari has received Orphan Drug designation in the U.S., Orphan Medicinal Product designation in the EU and Fast Track designation from the FDA.
+myBinderRelated Content
-
Transfusion Medicine, An Issue of Hematology/Oncology Clinics of North AmericaBlood transfusions are an important part...
-
Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in children with sickl...Although allogeneic haematopoietic stem ...
-
NIH Launches new Collaboration to Develop Gene-Based Cures for Sickle Cell Disease and HIV on Global ScaleThe National Institutes of Health plans ...
-
What is a Bone Marrow Transplant?https://www.youtube.com/watch?v=wrfT5wqH...
-
Gene therapy targets sickle-cell diseaseElliott Vichinsky estimates that at leas...
-
Improving the Lives of People with Sickle Cell Diseasehttps://www.youtube.com/watch?v=NPaV0glX...
-
Mount Sinai receives NIH grant to study use of inhaled corticosteroids for sickle cell treatmentThe Departments of Emergency Medicine an...
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.
