‘Best Biotech’ Prix Galien USA Awarded to GBT’s Oxbryta

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by Steve Bryson, PhD |

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GBT's Oxbryta wins 'Best Biotech' Prix Galien USA Award/Sickle Cell Anemia News/speaker at podium illustration

Oxbryta (voxelotor), Global Blood Therapeutics’ first-in-class oral therapy for sickle cell disease (SCD), has won the 2021 Prix Galien USA Award for best biotechnology product.

The award is among the health industry’s most prestigious honors, and recognizes an outstanding product that has improved patient well-being and impacted human health.

Proven to stop the sickling and destruction of red blood cells in SCD, Oxbryta was selected from a pool of 19 candidates by a panel of 12 academia and industry members — which includes five Nobel Prize winners.

“The Prix Galien Foundation’s recognition of Oxbryta is a testament to the importance of this innovation for a patient community that has suffered from limited treatment options for far too long,” Ted W. Love, MD, president and CEO of Global Blood Therapeutics (GBT), said in a press release.

“With the potential to modify the course of this disease, we are proud that Oxbryta is able to help many people with SCD today and in the future,” Love said.

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Oxbryta previously had been awarded industry honors, including the 2020 National Organization for Rare Disease (NORD) Rare Impact Award for Industry Innovation and the 2020 National Xconomy Award for Breakthrough Drug of the Year.

It is the first approved therapy that directly suppresses the underlying biological mechanism of SCD, which is caused by mutations in the HBB gene that encodes hemoglobin. This protein carries oxygen in red blood cells, and when mutated, tends to form clumps, or polymerize, leading to the sickling and destruction of red blood cells.

The sickling process results in hemolytic anemia — low hemoglobin levels due to red blood cell destruction — that compromises oxygen transport and delivery to tissues and organs throughout the body. By blocking hemoglobin polymerization, Oxbryta improves oxygen delivery throughout the body and potentially modifies the course of SCD, according to the company.

In 2019, the therapy received accelerated approval in the U.S. to treat SCD patients ages 12 and older. As a condition of this approval, GBT is currently evaluating Oxbryta’s ability to lower stroke risk in up to 224 SCD children, ages 2–15, in the placebo-controlled Phase 3 HOPE-KIDS 2 study (NCT04218084).

Due to the critical need for SCD treatments, Oxbryta has received various designations in the U.S. that supported and accelerated its development. These included breakthrough therapy, fast track, orphan drug, and rare pediatric disease designations. The therapy also has been granted promising innovative medicine designation in the U.K., and has been named an orphan drug and a priority medicine in Europe.

The European Medicines Agency is currently reviewing GBT’s application, which seeks full marketing approval of Oxbryta in Europe for SCD patients, ages 12 and older.

In the U.S., GBT also is asking regulators to expand the therapy’s use to treat SCD in children as young as 4. The U.S. Food and Drug Administration is currently reviewing the company’s request under priority review. A decision is expected no later than Dec. 25.

“Receiving the prestigious Prix Galien Award is a tremendous honor,” Love said of the award. “We are grateful to all the patients, their families, and physicians, as well as the entire GBT team for their tireless dedication in making this recognition possible.”