DISCLAIMER
The information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.compose your message
message sent
email sent successfully
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.”


Related Content
-
videos & visualsWhat Should Teens Ask Their Doctors About Hydroxyureahttps://www.youtube.com/watch?v=VZn15uXQ...
-
education & researchA Clinically Meaningful Fetal Hemoglobin Threshold for Children With Sickle Cell Anemia During Hydroxyurea TherapyHydroxyurea has proven clinical benefits...
-
education & researchLosartan for the nephropathy of sickle cell anemia: A phase-2, multicenter trialNephropathy is a common and progressive ...
-
news & eventsFDA grants CRISPR gene therapy fast track designation for sickle cell diseaseThe FDA granted fast track designation t...
-
news & eventsSickle Cell Treatment Endari Now Available in the United StatesEmmaus Life Sciences has announced that ...
-
news & eventsSickle Cell Foundation Nigeria, Rhieos Develop 1st Multi-SCD Registry in NigeriaIn a bid to improve quality of care for ...
-
videos & visualsTreatment Advances in Sickle Cell Disease, Amber M. Yates, MDhttps://www.youtube.com/watch?v=oFIOKC4r...
send a message
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.
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.