GBT To Submit MAA for Oxbryta for Sickle Cell Disease

Global Blood Therapeutics (GBT) will submit their Marketing Authorization Application (MAA) for Oxbryta to the EMA by 2021, says a recent BioSpace press release. Ultimately, the MAA will seek regulatory approval for Oxbryta to treat hemolytic anemia in patients over 12 with sickle cell disease. The goal is to fulfill unmet needs in SCD treatment for the estimated 52,000 European patients.

Sickle Cell Disease (SCD)

Sickle cell disease (SCD) consists of a group of hematologic disorders characterized by deformed red blood cells. These result from a mutation on the hemoglobin-producing gene. When sickle-shaped cells get caught along blood vessel walls, they block blood flow. The most common and severe form of SCD is sickle cell anemia.

Symptoms of SCD include:

  • Jaundice (yellowing skin and eyes)
  • Fatigue
  • Swelling of the hands and feed
  • Hemolytic anemia
  • Stroke
  • Bodily pain
  • Delayed growth
  • Brain, eye, spleen, liver, kidney, lung, heart, bone, join, and skin damage

Learn more about SCD.


For 9 years, Global Blood Therapeutics has worked to inform research and treatment for SCD. Their orally-administered therapy Oxbryta (voxelotor) inhibits a process called hemoglobin polymerization which causes red blood cell damage and malformation. In turn, this prevents hemolytic anemia: low hemoglobin levels resulting from red blood cell destruction. This form of anemia prevents enough oxygen from getting to organs and tissue throughout the body.

Included in the MAA are data from two clinical trials: Phase 2 HOPE-KIDS 1 and Phase 3 HOPE. Over the past few months, Oxbryta also received:

  • Accelerated approval from the FDA
  • Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations from the FDA
    • Some of these designations come with perks such as exclusive marketing, tax credits, and increased communication with the FDA.
  • Priority Medicines (PRIME) designation from the European Medicines Association (EMA)
  • Orphan Medicinal Product status from the European Commission (EC)

What are your thoughts about this new treatment? Share your stories, thoughts, and hopes with the Patient Worthy community!

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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