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
-
Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA u...Advances in allogeneic hematopoietic cel...
-
Ironwood Pharmaceuticals Announces FDA Orphan Drug Designation for Olinciguat for the Treatment of Sickle Cell Disea...Ironwood Pharmaceuticals, Inc., a commer...
-
CTX001 Continues to Show Promise in Severe SCDA single dose of CTX001, an experimental...
-
A single center experience of GBT440 treatment of severe anemia in sickle cell diseaseBackground: GBT440 is an oral, once-dail...
-
Gene therapy targets sickle-cell diseaseElliott Vichinsky estimates that at leas...
-
Pioneering a New Therapy for Sickle Cell DiseaseThe science behind sickle cell disease (...
-
Phase 3 Trial Recruiting to Test Rivipansel for Vaso-Occlusive Crisis in SCDGlycoMimetics, in collaboration with Pfi...
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.