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FSCDR’s Experience With Voxelotor (Gbt440) Treatment in Patients With Severe Sickle Cell Disease

ATLANTA — Lanetta Bronté, M.D., M.P.H., M.S.P.H., founder and chief health officer of the Foundation for Sickle Cell Disease Research (FSCDR) in Hollywood, Fla., presented results from seven adult sickle cell disease patients with severe anemia and multiple co-morbidities who were not eligible to participate in Global Blood Therapeutics’s (GBT) Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study (abstract #3545), at the 59th American Society of Hematology Meeting & Exposition on Monday evening.

“Patients with severe SCD, who often suffer life-threatening complications, are refractory to conventional therapy and, thus, are very difficult to manage because treatment options are very limited,” said Dr. Bronté. The patients, four women and three men, age 22 to 67, received voxelotor for six to 17 months through single-patient compassionate access and monitored over 24 weeks at Dr. Bronté’s outpatient center. Results showed:

“We are grateful that these seven patients at our center were able to receive voxelotor through compassionate access and are so pleased with the clinical improvements we saw on meaningful endpoints,” said Dr. Bronté.

FSCDR is a leading innovator in sickle cell disease. Dr. Bronté opened the center in Nov. 2014 to address the growing trend of fragmented care and an increase in emergency room visits and inpatient utilization in adolescents and adults with SCD. FSCDR runs the U.S.’s only outpatient center solely devoted to sickle cell care and services and is independent from any hospital system or academic institution.

Compassionate access is an option facilitated by the U.S. Food and Drug Administration (FDA) to make available, prior to regulatory approval, investigational medicines for the treatment of serious or life-threatening disease or conditions for which there are no ongoing clinical trials and there is a lack of satisfactory therapeutic alternatives. Voxelotor is an investigational treatment and controlled clinical trials are needed to confirm the clinical benefits and safety seen in the case study of seven patients.

You can read the full poster, here.

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