Clinical Trial Updates (SCD)

Oxbryta (Voxelotor)

Update: 30 June 2021

A new analysis of data from 45 children with SCD, aged 4 to 11 years, enrolled in the open-label Phase 2a HOPE-KIDS 1 Study (GBT440-007) showed that treatment with Oxbryta (1,500 mg or weight-based equivalent dispersed in a pediatric-appropriate formulation) resulted in rapid and sustained improvements in hemoglobin.

An increase in hemoglobin of greater than 1 g/dL from baseline was observed in 47% of patients as early as two weeks and sustained through 24 weeks, consistent with results in patients ages 12 years and older in the Phase 3 HOPE Study. Concurrent improvements in markers of hemolysis were also observed. Relevant data was presented at EHA2021.



Update: 08 January 2021

No update available


Update: 05 November 2020

72-week analysis of the Phase 3 HOPE Study of Oxbryta® (voxelotor) tablets will be presented at ASH2020 in December 2020.



Update: 25 August 2020

The European Medicines Agency (EMA) has granted Oxbryta® Priority Medicines (PRIME) designation, and the European Commission (EC) designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD.

The company plans to submit a Marketing Authorization Application (MAA) to EMA to treat hemolytic anemia in sickle cell disease (SCD) patients ages 12 years and older by mid-2021.

The planned MAA will include data from the Phase 3 HOPE Study and the Phase 2 HOPE-KIDS 1 Study, both of which enrolled patients at clinical sites in Europe.

The company also intends to initiate an Early Access Program in Europe for patients and physicians who may need access to Oxbryta® prior to potential marketing authorization.

USA: Discussions for the potential use of Oxbryta® (voxelotor) for the treatment of sickle cell disease (SCD) in children ages 4 to 11 years are underway under the FDA’s accelerated approval pathway.



Update: 31 May 2020

  • A retrospective analysis of the HOPE 3 trial will be presented at EHA25 Congress in June.
  • Access in USA slowed by COVID-19 pandemic and social distancing.



Update: 31 March 2020

No update available.


Update: 31 January 2020

In a draft report, the Institute for Clinical and Economic Review (a cost watchdog) concluded that the newly authorised sickle cell disease drugs in the US (from GBT, Novartis and Emmaus Medical) are too expensive to meet traditional cost-effectiveness measures. To fall within one measure of cost-effectiveness—below $150,000 per quality-adjusted life year—the companies would have to dramatically cut their prices.

For GBT’s Oxbryta, the cut would have to be almost 90% – a cost of $9,218 per year would be more appropriate —down from an estimated list price of $84,000 per year. And for Novartis’ Adakveo, ICER’s calculations suggested an annual cost of $25,410, less than one-third of its existing cost of $88,000 per year, as estimated by ICER.


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