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GBT Initiates Early Access Program for Voxelotor in Patients with Sickle Cell Disease in Europe and Other Regions Outside the United States

SOUTH SAN FRANCISCO, Calif., Dec. 02, 2020 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced the initiation of an early access program for voxelotor, in Europe and other regions outside the United States, for the treatment of hemolytic anemia in sickle cell disease (SCD) patients ages 12 years and older.

“With no currently approved therapies outside the United States to treat hemolytic anemia in sickle cell disease, the unmet need in this devastating disease is profound,” said Ted W. Love, M.D., president and chief executive officer of GBT. “As part of our deep, long-term commitment to patient access worldwide, we are pleased to initiate this early access program for eligible patients who may benefit from voxelotor.”

A first-in-class oral, once-daily therapy, voxelotor directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction), which impairs adequate oxygen delivery to the tissues and organs in the body.

Voxelotor is approved in the United States under the trade name Oxbryta® for the treatment of SCD in patients ages 12 years and older. GBT previously announced it plans to seek regulatory approval to treat hemolytic anemia in SCD in patients ages 12 years and older in Europe.

An early access program is a mechanism to make medicines available pre-approval upon request by a physician for appropriate patients with no alternative treatment option.

Through the voxelotor early access program, physicians in countries with an early access regulatory and legal pathway may be able to request voxelotor for eligible SCD patients who do not have access to the medicine as part of a clinical trial.

The program is being implemented in partnership with and administered by Inceptua Group’s Medicines Access division, which has expertise in the strategy, design, and operational implementation of pre-approval access programs that make pharmaceutical products in clinical development available to patients as appropriate.

All requests must be submitted by the treating physician on behalf of the eligible SCD patient. Healthcare providers can obtain details about the voxelotor early access program by contacting Inceptua at

For the six Middle Eastern countries in the Gulf Cooperation Council (GCC), GBT previously announced an exclusive distributorship that references the U.S. approval of Oxbryta to allow for access to the medicine while health authorities conduct their regulatory reviews, thereby obviating the need for this new early access program.

About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in the United States,1 an estimated 52,000 people in Europe,2 and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.1 It also affects people of Hispanic, South Asian, Southern European, and Middle Eastern ancestry.1 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.3 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.3-5 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.4-7

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. With the potential to improve hemolytic anemia and oxygen delivery, GBT believes that Oxbryta has the potential to modify the course of SCD. On November 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.8 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.

GBT plans to seek regulatory approvals to expand the potential use of Oxbryta in the United States for the treatment of SCD in children ages 4 to 11 years and to treat hemolytic anemia in SCD in patients ages 12 years and older in Europe.

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Last Updated: 02-Dec-2020