Google
Trusted Resources: News & Events
Latest announcements and gatherings
L-glutamine Oral Powder Significantly Reduces Acute Complications of Sickle Cell Disease
Approved by the US Food and Drug Administration (FDA) in July 2017 to reduce the acute vaso-occlusive pain complications of sickle cell disease, L-glutamine oral powder (Endari) was the first drug approved for the rare blood disease in the 20 years since the approval of hydroxyurea.
Now, the New England Journal of Medicine (NEJM) has published the 48-week phase 3 clinical trial results that supported the FDA approval. From the data acquired in the phase 3 trial, investigators concluded that the median number of pain crises in children and adults with sickle cell disease was lower among those who received oral therapy with l-glutamine, administered alone, or with hydroxyurea, compared with those who received placebo, with or without hydroxyurea.
“The idea that this [publication] comes a year after [approval] really reflects the vigor of the review process and shows NEJM really did its job of assuring the public interest that L-glutamine, along with the findings of the study, are real and significant,” Darrell W. Harrington, MD, MACP, chief medical officer at Emmaus Life Sciences, told Rare Disease Report®.
+myBinderRelated Content
-
Emmaus, a leader in sickle cell disease treatment, signs agreement with Cardinal Health to solidify distribution net...Emmaus Life Sciences, Inc. announces...
-
Sickle cell patient with severe anemia rapidly improves with voxelotor, case study showsGlobal Blood Therapeutics (GBT) reported...
-
Endari: A Treatment Option for Sickle Cell DiseaseEndari is an amino acid, approved by Foo...
-
Emmaus, a leader in sickle cell disease treatment, signs agreement with McKesson to expand distribution network for ...Emmaus Life Sciences, Inc., anno...
-
Orphan Drugs for Sickle Vaso-occlusion: Dawn of a New Era of Targeted TreatmentWhile an orphan disease in the USA, sick...
-
Global Blood Therapeutics Announces Completion of Safety Review by Independent Data and Safety Monitoring Board (DSM...Global Blood Therapeutics, Inc. (GBT) (N...
-
Gamida Cell Announces $3.5 Million Grant from the Israeli GovernmentGamida Cell, a leader in cellular and im...
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.