Case Study
April 5, 2021

Delivering Innovative Treatment To Underserved Patients

Global Blood Therapeutics, Inc. (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, with a focus on sickle cell disease (SCD).

SCD is a hereditary, lifelong rare blood disorder that alters hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body. SCD affects roughly 100,000 individuals in the U.S., and approximately 52,000 people in Europe. There are more than 350,000 people living with SCD in the U.S., Europe, the Gulf Cooperation Council (GCC) region in the Middle East and Latin America combined.

Worldwide, there are millions of people living with SCD, which occurs predominately in populations of African, Middle Eastern and South Asian ancestry and has an estimated global incidence of 250,000 to 300,000 births annually.

In those living with SCD, hemoglobin polymerization causes red blood cells to become sickled — deoxygenated, crescent-shaped and rigid — causing hemolytic anemia and blockages which impede the flow of blood and oxygen throughout the body. Hemolytic anemia is low hemoglobin due to red blood cell destruction.

GBT, which was founded in 2011 and went public in 2015, provides a medicine known as Oxbryta® (voxelotor) tablets, which was approved by the U.S. Food and Drug Administration (“FDA”) in 2019 to treat patients with sickle cell disease who are at least 12 years old.

Discovered by scientists at GBT, Oxbryta is an oral, once-daily therapy that directly inhibits hemoglobin polymerization, which is the root cause of the sickling and destruction of red blood cells seen in SCD.

In 2021, GBT plans to submit for FDA approval for Oxbryta for pediatric SCD patients ages 4 to 11 years. In the U.S., GBT has started a multi-center expanded access protocol for these pediatric SCD patients to enable access to Oxbryta treatment prior to potential approval. 

Beyond its focus on the U.S., GBT is also working to expand access for Oxbryta in Europe, the Middle East, and other regions of the world.

In late January 2021, GBT announced that the European Medicines Agency (EMA) commenced review of GBT’s Marketing Authorization Application (MAA) for Oxbryta, which if approved will provide approval to market Oxbryta as a treatment for patients with hemolytic anemia.

In late 2020, GBT started an early access program in Europe and other regions outside the U.S., for the treatment of hemolytic anemia in SCD patients ages 12 years and older. Through this program, physicians in countries with an early access regulatory and legal pathway can request voxelotor for eligible SCD patients who are unable to participate in GBT’s clinical trials. In addition, in the GCC, GBT has entered into an exclusive arrangement to distribute Oxbryta in the region. 

Beyond Oxbryta, GBT is also engaged in other research and development activities, including working on new targets to potentially develop next generation of treatments for SCD, including inclacumab, a P-selectin inhibitor, which is a clinically validated target in SCD, known to reduce the incidence of vaso-occlusive crises (VOCs), and GBT’s next generation hemoglobin polymerization inhibitor, GBT021601 (GBT601).

“Sickle cell disease is a devasting blood disorder that destroys lives and ends them far too soon. Since our founding ten years ago, GBT has been a company dedicated to meeting the needs of these underserved patients through groundbreaking science, community support and a strong company culture,” CEO Ted W. Love, M.D., said. “We’re very pleased with our progress to date on providing access to Oxbryta to thousands of SCD patients, and we’re excited about the recent advances we’ve made on our R&D pipeline as we continue our on our journey to transform SCD into a well-managed condition.”

Global Blood Therapeutics At A Glance

  • Publicly traded company (NASDAQ: GBT)
  • Founded in 2011
  • Headquartered in South San Francisco, California (USA)
  • Goodwin Life Sciences client since 2011
  • 389 Employees