GBT Initiates Expanded Access Program for Voxelotor in Patients With Sickle Cell Disease in Brazil
Excerpt from the Press Release:
SOUTH SAN FRANCISCO, Calif., July 14, 2022 (GLOBE NEWSWIRE) — Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced the initiation of an expanded access program (EAP) for voxelotor in Brazil for the treatment of sickle cell disease (SCD) in adult and pediatric patients 12 years of age and older who cannot satisfactorily be treated with a medicine authorized for use in Brazil. ANVISA, the Brazilian Health Regulatory Agency, has authorized the voxelotor EAP, which is actively enrolling at clinical sites around the country.
“There are up to 100,000 people estimated to have sickle cell disease in Brazil, yet there are no currently approved treatments that directly address the underlying cause of the disease,” said Kim Smith-Whitley, M.D., executive vice president and head of research and development at GBT. “As part of our deep, ongoing commitment to patient access worldwide, we are pleased to initiate this expanded access program for eligible patients in Brazil who may benefit from voxelotor, which we believe can potentially modify the course of this debilitating disease.”
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 anemia and hemolysis (low hemoglobin due to red blood cell destruction), which impairs adequate oxygen delivery to tissues and organs in the body. Voxelotor is not currently approved for marketing by ANVISA in Brazil.
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