Patients treated with voxelotor achieved a statistically significant increase in hemoglobin compared to baseline, the primary endpoint, at 12 weeks.
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.
This designation demonstrated the FDA’s recognition that the SCD community faces a critical need for new treatments and further endorsed the potential of GBT’s voxelotor program.
Voxelotor became the first potential treatment for SCD to be accepted for the EU’s new 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 enrollment was launched with plans to conduct the study at approximately 80 leading SCD sites globally. The study is expected to enroll up to approximately 400 adults and adolescents with SCD.
In growing recognition of the current limitations in treatment for SCD among the global regulatory community, the EMA designated voxelotor as an orphan medicinal product, qualifying GBT for a range of development and commercial incentives in the European Union (EU).
The FDA approved the first of its kind pivotal trial design for SCD that combines a Phase 2b dose escalation phase with a Phase 3 efficacy phase, to accelerate voxelotor’s development and completion of this registrational study.
With orphan drug designation, GBT qualified for various incentives for voxelotor, including tax credits for qualified clinical trials and market exclusivity upon U.S. marketing approval.
Acknowledging that SCD is a serious health problem that needs new treatment options, the FDA granted voxelotor Fast Track designation which has the potential to accelerate drug approval and access by patients.
GBT raised $120 million in the initial public offering to fund further clinical trials of voxelotor. Since its IPO, GBT has successfully completed multiple follow-on offerings, most recently in March 2018.
Within 15 months of identifying voxelotor as a potential clinical candidate, the first eight patients were dosed in a Phase 1/2 clinical trial for SCD.
Experiments run in collaboration with Emory University provided pre-clinical proof-of-concept for voxelotor as a polymerization inhibitor.
Inspired by the promise of GBT’s science and the plight of SCD patients, board member Ted Love came out of retirement to become CEO.
Voxelotor Named Clinical Candidate
Based on promising pre-clinical data, voxelotor was nominated as the first clinical candidate of GBT.
GBT440 Disclosed in a U.S. Patent Application
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 initially undertook a traditional medicinal chemistry approach for drug discovery to identify promising molecules.