Global Blood Therapeutics, Inc. announced the initiation of an expanded access program for voxelotor in Brazil for the treatment of sickle cell disease 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. 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, which impairs adequate oxygen delivery to tissues and organs in the body. Voxelotor is not currently approved for marketing by ANVISA in Brazil. An expanded access program is a mechanism to make medicines available pre-approval upon request by a physician for eligible patients with no alternative treatment option.

ANVISA's authorization of an EAP is based on the following criteria: The product is intended for patients with a serious, debilitating, and/or life-threatening disease. There is no satisfactory therapeutic alternative with products registered in Brazil. The authorization to use the product is delivered based on the request, and only under the responsibility of the prescribing physician, as it is expected to deliver a significant benefit to the patient.

Unr the e voxelotor EAP, voxelotor will be supplied free of chargeor as long as the patient requires treatment. This 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 available to patients as appropriate in multiple countries around the world.