Global Blood Therapeutics has secured accelerated approval from the US Food and Drug Administration (FDA) for the use of Oxbryta (voxelotor) to treat sickle cell disease (SCD) patients aged 12 years and above.

Oxbryta is the first approved drug that addresses the primary cause of SCD by directly inhibiting sickle haemoglobin polymerisation.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

SCD is a blood disorder, where a genetic mutation leads to the formation of abnormal haemoglobin called sickle haemoglobin via the haemoglobin polymerisation process.

FDA approval of the drug comes from positive results of Phase III HOPE clinical trial conducted in 274 patients.

During the study, 90 patients were treated with Oxbryta 1,500mg, while 92 were administered 900mg. The remaining 92 were given a placebo.

After 24 weeks of therapy, 51.1% of participants on the drug experienced higher than 1g / dL rise in haemoglobin versus 6.5% on placebo.

Common drug-related side effects were a headache, diarrhoea, abdominal pain, nausea, fatigue, rash and fever.

FDA acting commissioner Brett Giroir said: “Today’s approval provides additional hope to the 100,000 people in the US, and the more than 20 million globally, who live with this debilitating blood disorder.”

The company must continue the assessment of Oxbryta in the HOPE-KIDS 2 study as a condition of accelerated approval.

HOPE-KIDS 2, set to launch by the end of the year, is a post-approval confirmatory study, which will use transcranial doppler (TCD) flow velocity to measure a decrease in stroke risk in children aged two to 15 years.

Global Blood Therapeutics intends to make the drug commercially available by next month, priced at $10,417 per month or approximately $125,000 per year.

Last week, the FDA approved Novartis’ Adakveo (crizanlizumab) to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in SCD patients aged 16 years and above.