Google
Trusted Resources: News & Events
Latest announcements and gatherings
FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe SCD and Transfusion-Dependent Beta Thalassemia
BOSTON & ZUG, Switzerland–(BUSINESS WIRE)–Jun. 8, 2023– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively. Updated data from the pivotal trials supporting the regulatory submissions will be presented at the Annual European Hematology Association Congress on June 11, 2023.
+myBinderRelated Content
-
Global Blood Therapeutics (GBT) announces upcoming data presentations supporting Voxelotor SCD programGlobal Blood Therapeutics, Inc. (GBT) ...
-
Dexmedetomidine as an adjuvant to analgesic strategy during vaso-occlusive episodes in adolescents with sickle cell ...Sickle cell disease (SCD)-patients exper...
-
MARAC Statement: Crizanlizumab (Adakveo)July 7, 2023 - SCDAA’s Medical and Res...
-
FDA Awards UNC Researcher $2M Grant to Study Kidney Disease in Sickle Cell AnemiaThe U.S. Food and Drug Administration (F...
-
NHLBI Stepping Up Efforts to Apprise SCD Patients of Therapies and TrialsWide interest in a CBS 60 Minutes story ...
-
MaxCyte and U.S. National Institutes of Health’s National Heart, Lung, and Blood Institute enter cooperative r...MaxCyte, Inc. today announces it has e...
-
The FDA Encourages New Treatments for Sickle Cell DiseaseThe FDA continues to work with stakehold...
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.