GBT Initiates Expanded Access Protocol for Oxbryta® (voxelotor) in Pediatric Patients with Sickle Cell Disease in the United States

IndustryPRwire -- Global Blood Therapeutics announced that an expanded access protocol (EAP) for Oxbryta® (voxelotor) in pediatric patients with sickle cell disease (SCD) has been initiated and is currently enrolling. The EAP is designed to provide access to Oxbryta prior to approval for children ages 4 to 11 years with SCD in the United States who have no alternative treatment options and are ineligible to participate in clinical trials of Oxbryta. GBT enrolled its first patient in the EAP in January 2021.

“Early intervention and treatment of sickle cell disease are critical and have shown potential to modify the course of this devastating disease. Despite this, children under the age of 12 have limited approved therapeutic options,” said Ted W. Love, M.D., president and chief executive officer of GBT. “As part of our commitment to patient access for Oxbryta, we are pleased to initiate this expanded access protocol as we work towards our planned pediatric approval submission for the potential use of Oxbryta in this age group.”

An oral, once-daily therapy, Oxbryta is the first and only approved therapy that directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. Oxbryta is approved in the United States for the treatment of SCD in patients ages 12 years and older. GBT previously announced plans to submit a New Drug Application (NDA) for a pediatric formulation of Oxbryta for the treatment of SCD in children ages 4 to 11 years by mid-2021.

The EAP allows access to Oxbryta for pediatric patients 4 to 11 years of age who have an urgent medical need, are unable to participate in clinical studies, and do not have other treatment options based on their physician’s judgment. The study protocol is open-label and includes new age-appropriate formulations of Oxbryta, including the powder for oral suspension that will be included in GBT’s planned NDA.

There are multiple clinical sites across the United States participating in the treatment protocol. For more information on the location of participating sites, protocol and eligibility criteria, please visit [Identifier: NCT04724421]. Additional information can be found at the GBT Medical Information web portal: or by calling 1-833-GBT-4YOU (1-833-428-4968).

About Sickle Cell Disease

Sickle cell disease (SCD) affects an estimated 100,000 people in the United States, an estimated 52,000 people in Europe, and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa. It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry. SCD is a lifelong inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body. Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled – deoxygenated, crescent-shaped and rigid. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.

About Oxbryta® (voxelotor) Tablets

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, GBT believes Oxbryta blocks polymerization and the resultant sickling and destruction of red blood cells, which are primary pathologies faced by every single person living with SCD. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD. On Nov. 25, 2019, Oxbryta received U.S. Food and Drug Administration (FDA) accelerated approval for the treatment of SCD in adults and children 12 years of age and older.

As a condition of accelerated approval, 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 15 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 has been granted Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), and the European Commission (EC) has designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD.

The EMA has accepted for review GBT’s Marketing Authorization Application (MAA) seeking full marketing authorization of Oxbryta in the European Union to treat hemolytic anemia in SCD patients ages 12 years and older. GBT also plans to seek regulatory approval to expand the potential use of Oxbryta in the United States for the treatment of SCD in children as young as 4 years old.

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