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
-
Aisha Walker, PhDDr. Walker is a Research Assistant Profe...
-
Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in...Importance: In children with sickle cel...
-
Gene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
-
The role of nutrition in the pathophysiology and management of sickle cell disease among children: A review of liter...Sickle cell disease (SCD) is one of the ...
-
CRISPR could end sickle cell disease, but signing up black patients for clinical trials will be a hard sellThe first attempts to use a groundbreaki...
-
Global Sickle Cell Disease Treatment Market 2019–2023| Evolving Opportunities with ADDMEDICA and Bristol-Myers Squ...The global sickle cell disease treatment...
-
Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Diseasehttps://www.youtube.com/watch?v=CWTW5pDP...
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
SomeBodyToTalkTo connects people affected by rare diseases with trusted resources, supportive communities, and opportunities to share their experiences.