Global Blood Therapeutics Announces FDA Approval of Oxbryta (voxelotor) for the Treatment of Sickle Cell Disease in Patients Aged 12 and Older

VCBeat (WeChat ID: vcbeat) learned from foreign media reports that on November 25, 2019 (local time), the U.S. Food and Drug Administration (FDA) approved Global Blood Therapeutics’ (GBT) new drug, Oxbryta, for the treatment of pediatric and adult patients aged 12 years and older with sickle cell disease (SCD).

Oxbryta was approved through the FDA’s Priority Review pathway, with just over two months elapsing between the acceptance of its New Drug Application and final approval. Previously, the FDA had granted Oxbryta Breakthrough Therapy designation, Fast Track designation, Orphan Drug designation, and Rare Pediatric Disease designation for the treatment of sickle cell disease (SCD). In Europe, the European Medicines Agency (EMA) has granted Oxbryta PRIME (PRIority MEdicines) designation and Orphan Drug designation for the treatment of SCD.

GBT, founded in 2012 and headquartered in San Francisco, USA, is a clinical-stage biopharmaceutical company. The company is dedicated to discovering, developing, and delivering innovative therapies, bringing hope to underserved patient populations.

Oxbryta, developed by Global Blood Therapeutics, is the first approved therapeutic agent that directly inhibits sickle hemoglobin polymerization. The active pharmaceutical ingredient in Oxbryta is voxelotor (formerly known as GBT440), which functions by increasing the oxygen affinity of hemoglobin. Since oxygenated sickle hemoglobin does not polymerize, voxelotor blocks polymerization and the subsequent sickling and destruction of red blood cells. Voxelotor can improve hemolytic anemia and oxygen transport, and has the potential to modify the course of sickle cell disease (SCD).

Sickle cell disease (SCD) is a lifelong genetic blood disorder that causes red blood cells to assume an abnormal shape, restricting blood flow within the vessels and leading to severe pain and organ damage.

“Oxbryta inhibits the polymerization of deoxy-sickle hemoglobin, which is the key abnormality in SCD,” said Dr. Richard Pazdur, Director of the FDA’s Office of Oncologic Diseases. “By preventing red blood cells from assuming a sickle shape and aggregating, Oxbryta prevents the decline in hemoglobin levels caused by hemolysis. It provides a new treatment option for patients suffering from this serious, life-threatening disease.”

Ted W. Love, President and Chief Executive Officer of GBT, stated, “Today’s approval is not only a significant milestone for GBT, but also a milestone for patients with sickle cell disease (SCD) and their families. Eight years ago, we began collaborating with the SCD community to transform the treatment landscape for this devastating lifelong condition. We are proud to bring this breakthrough therapy to the SCD community.”

During a conference call with investors, David Johnson, Chief Commercial Officer of GBT, stated that the drug is priced at $10,417 per month, with 65% of insured patients paying a net price of approximately $8,000 per month after discounts. GBT executives noted that approximately 50% of patients with sickle cell disease (SCD) are covered by Medicare, 30% have basic commercial insurance, and 10–15% have dual coverage through both Medicare and commercial insurance. The company plans to keep the drug’s price unchanged for the next three years and will also launch a patient support program called GBT Source, which means eligible patients will not incur any out-of-pocket costs.

(Compiled by Zhao Siyun)