Oxbryta Granted Marketing Approval in Great Britain

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by Steve Bryson, PhD |

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Oxbryta (voxelotor) has been granted marketing approval in Great Britain to treat hemolytic anemia in people ages 12 and older with sickle cell disease (SCD).

Eligible patients may take the once-daily oral therapy alone or in combination with hydroxyurea, a standard SCD therapy.

The approval was granted by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to Global Blood Therapeutics (GBT), which developed the therapy.

“We welcome the MHRA’s marketing authorization of [Oxbryta] as a new treatment option for many people in Great Britain living with sickle cell disease, a devastating life-long condition which for far too long has seen little therapeutic innovation,” Beatriz Pujol, PhD, vice president at GBT, said in a press release.

In the U.S., Oxbryta was granted accelerated approval in 2019 for SCD patients 12 and older, which then was extended to include children as young as 4 using a more suitable formulation for young children.

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More recently, the European Commission approved the therapy, alone or in combination with hydroxyurea, for those 12 and older. Oxbryta also is available for the same patient population in the United Arab Emirates.

Last year, the therapy was deemed a Promising Innovative Medicine by the MHRA, a label applied to promising treatments that are likely to impart significant benefits to U.K. patients. Then, earlier this year, the therapy was made available to eligible patients in the U.K. under MHRA’s early access program.

In SCD, abnormal clumping of hemoglobin, the protein that transports oxygen inside red blood cells, causes the cells to form a sickle-like shape, becoming more rigid and sticky. This leads to hemolytic anemia, marked by the destruction of red blood cells and less oxygen delivery to tissues.

Oxbryta is designed to treat the underlying cause of the disease by increasing hemoglobin’s ability to bind oxygen, thus preventing its clumping and red blood cell destruction.

The therapy aims to restore hemoglobin levels, red blood cell function, and oxygen delivery while alleviating the risk of vaso-occlusive crises — episodes of pain caused by sickled cells blocking blood flow.

The marketing approval by the MHRA is based on data demonstrating that Oxbryta treatment led to clinically meaningful improvements in hemoglobin levels and reduced hemolysis markers.

Results from the Phase 3 HOPE trial (NCT03036813), a worldwide study in 274 SCD patients ages 12 to 65, showed that 51% of the 90 patients receiving Oxbryta achieved a hemoglobin response after six months, compared with 7% with placebo.

Treatment also significantly reduced bilirubin and reticulocyte levels — two biomarkers of hemolysis. These benefits were seen regardless of combined hydroxyurea use.

More Oxbryta trials

The therapy also is being tested in the Phase 2 HOPE-KIDS 1 trial (NCT02850406), which is currently recruiting up to 155 SCD children, ages 6 months to 17 years, in the U.S., the U.K., and Lebanon. Six-month data from the first 45 treated children, ages 4–11, were similar to those in older HOPE participants.

Also underway is the HOPE-KIDS 2 study (NCT04218084). The trial is testing the therapy against a placebo in up to 224 children, ages 2–14, with SCD.

GBT will now engage with the National Institute of Health and Care Excellence and the Scottish Medicines Consortium to make the therapy available to patients in Great Britain, which encompasses England, Wales, and Scotland.

“Following this marketing authorization by the MHRA, we look forward to working with the National Institute of Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) with the goal of helping to facilitate rapid access to [Oxbryta] for people living with sickle cell disease who may benefit from this important treatment,” Pujol said.