Pfizer Inc. has announced the completion of its acquisition of Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities starting with sickle cell disease (SCD). The acquisition reinforces Pfizer’s commitment to SCD, building on a 30-year legacy in the rare hematology space.
acquisition of Global Blood Therapeutics brings a portfolio and pipeline that has the potential to address the full spectrum of critical needs for this underserved community. GBT discovered and developed Oxbryta (voxelotor), a first-in-class medicine that directly targets the root cause of SCD. In addition, GBT’s promising pipeline of preclinical and clinical investigational assets focused in SCD includes GBT021601 (GBT601) and inclaclumab, both of which have received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA).
“With Global Blood Therapeutics’ talent, portfolio, and pipeline now part of Pfizer, we look forward to accelerating innovation and expeditiously bringing multiple potential best-in-class treatments to people living with sickle cell disease,” said Aamir Malik, Chief Business Innovation Officer, Executive Vice President, Pfizer. “In line with our value of equity, Pfizer is committed to addressing the underserved needs of the sickle cell disease community. We are excited about these potential breakthroughs and the opportunity to transform the lives of these patients.”
SCD is a lifelong, devastating inherited blood disorder impacting millions of people worldwide, predominantly in populations of African, Middle Eastern and South Asian descent. Pfizer will continue to build on the companies’ shared commitment to and engagement with the SCD community.
Sickle Cell Disease
Sickle cell disease (SCD) is a lifelong, debilitating inherited blood disorder characterized by hemolytic anemia, acute pain crises and progressive end organ damage. Acute pain crisis, or vaso-occlusive crisis (VOC), occurs when sickled red blood cells irritate the lining of blood vessels and cause an inflammatory response leading to vascular occlusion, tissue ischemia and pain. Complications of SCD begin in early childhood and are associated with shortened life expectancy. Early intervention and treatment of SCD have shown potential to modify the course of this disease, reduce symptoms and events, prevent long-term organ damage, and extend life expectancy. Historically, there has been a high unmet need for therapies that address the root cause of SCD and its acute and chronic complications. SCD occurs particularly among those whose ancestors are from sub-Saharan Africa, though it also occurs in people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.
Oxbryta (voxelotor)
Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, Oxbryta inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and hemolytic anemia, which are primary pathologies faced by every single person living with SCD.
In November 2019, the FDA granted accelerated approval for Oxbryta tablets for the treatment of SCD in adults and children 12 years of age and older, and in December 2021, the FDA expanded the approved use of Oxbryta for the treatment of SCD in patients 4 years of age and older in the United States. As a condition of accelerated approval for patients ages 4 and older in the United States, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 14 years of age.
In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta received the prestigious 2021 Prix Galien USA award for “Best Biotechnology Product” from The Galien Foundation.
Oxbryta has been granted Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), Oxbryta was designated by the European Commission (EC) as an orphan medicinal product for the treatment of patients with SCD, and Oxbryta was granted Promising Innovative Medicine (PIM) designation in the United Kingdom from the Medicines and Healthcare products Regulatory Agency (MHRA). In February 2022, the European Commission (EC) granted Marketing Authorization for Oxbryta for the treatment of hemolytic anemia due to SCD in adult and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea). The MHRA has granted Oxbryta marketing authorization in Great Britain for the treatment of hemolytic anemia due to SCD in adult and pediatric patients 12 years of age and older. In addition, Oxbryta has received marketing authorization for the treatment of SCD in adults and children 12 years of age and older in the Gulf Cooperation Council countries of Kuwait, Oman and the United Arab Emirates (UAE).
Source: Pfizer
