GBT is working to discover, develop and deliver life-changing treatments for people living with grievous blood-based disorders, starting with sickle cell disease
Our goal is to transform the treatment of sickle cell disease (SCD), an overlooked rare condition, and help address the urgent needs of the community. Our mission is driven by the historical lack of understanding and attention given to SCD. Although the fundamental cause of SCD has been understood for decades, therapeutic innovation and access to care has significantly lagged.
GBT’s medicine is the first treatment approved by the FDA to target an underlying cause of SCD. We are developing additional investigational treatments that have the potential to better the lives of patients.
Our history: we’re proud of all we have accomplished alongside the SCD community in our first 10 years and remain committed to our goal of changing the treatment paradigm in SCD.
The approval of GBT’s medicine for SCD was expanded to treat children ages 4 years to less than 12 years. The FDA also approved Oxbryta tablets for oral suspension, a new dispersible tablet dosage form.
Considered the equivalent of the Nobel Prize in biopharmaceutical research, Prix Galien USA honored Oxbryta as the Best Biotechnology Product for 2021.
The GBT Foundation is a community-focused, charitable entity that seeks to improve the health and well-being of underserved patient communities around the world, particularly for people living with SCD.
Complete 72-week results from the Phase 3 HOPE Study were published in The Lancet Haematology.
GBT is seeking full marketing approval from the EMA for Oxbryta to treat hemolytic anemia in patients with SCD who are 12 years of age and older.
GBT is collaborating with Syros to discover new medicines that induce fetal hemoglobin to treat SCD and beta thalassemia.
GBT’s first medicine for SCD received accelerated approval in the U.S. three months prior to our PDUFA date.
Priority Review is granted to therapies that the FDA determines have the potential to provide a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious condition.
GBT’s Phase 3 HOPE Study of Voxelotor in SCD Published in The New England Journal of Medicine and Presented at the 24th European Hematology Association Congress
Topline results for all patients enrolled in the Phase 3 HOPE Study were published in The New England Journal of Medicine.
The ACCEL Grants Program provides funding to support the development of sustainable access to care programs for people with SCD in the U.S.
Breakthrough Therapy designation expedites the development and review of drugs that have demonstrated preliminary clinical evidence indicating the potential for substantial improvement over available therapy.
Voxelotor became the first potential treatment for SCD to be accepted for the EU’s new Priority Medicines (PRIME) designation, enabling frequent and early interactions with the EMA to accelerate evaluation and potential marketing approval.
The HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS
PolymErization) Study was designed to assess the efficacy of voxelotor
in producing meaningful clinical improvements in anemia.
GBT became a public company.
Within 15 months of voxelotor being identified as a potential clinical candidate, the first 8 patients were dosed in a Phase 1/2 clinical trial in SCD.
Voxelotor (GBT440) Discovered
GBT began testing GBT440 in 2012 to understand its mechanism of action. This molecule would later be named voxelotor.
Global Blood Therapeutics, Inc. (GBT), Is Founded
The company was founded to develop medicines that could make a transformative impact, and SCD was our first focus.