Google
Trusted Resources: News & Events
Latest announcements and gatherings
GBT Receives FDA Breakthrough Therapy Designation for Voxelotor for Treatment of Sickle Cell Disease (SCD)
Global Blood Therapeutics, Inc. (GBT) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to voxelotor (previously called GBT440) for the treatment of sickle cell disease (SCD). Voxelotor is being developed as a disease-modifying therapy for SCD and previously received European Medicines Agency (EMA) Priority Medicines (PRIME) designation for the treatment of SCD.
“The FDA’s decision to grant voxelotor the first Breakthrough Therapy designation for the treatment of sickle cell disease reflects a recognition of the promising efficacy and safety data we have collected to date for this investigational drug, as well as an acknowledgement of the overwhelming need for major advances over available therapies in the treatment of SCD patients,” said Ted W. Love, president and chief executive officer of GBT. “This designation is another significant milestone for GBT as we work to expedite the development of voxelotor.”
+myBinderRelated Content
-
In France, Boy Becomes First Sickle Cell Disease Patient to be Treated With Gene TherapyA 13-year-old boy with sickle cell disea...
-
Chronic Opioid Use Pattern in Adult Patients with Sickle Cell DiseaseBackground: Pain, the hallmark complicat...
-
A Conversation on Hydroxyurea: Insights and Information for the Sickle Cell Communityhttps://www.youtube.com/watch?v=Y6fmJn-5...
-
Endari: A Treatment Option for Sickle Cell DiseaseEndari is an amino acid, approved by Foo...
-
BCL11A-based gene therapy for sickle cell disease passes key preclinical testA precision-engineered gene therapy viru...
-
Manage Your Sickle Cell Disease Symptomshttps://www.onescdvoice.com/wp-content/u...
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.