Pediatric patients who are eligible for participation in this EAP will be treated with voxelotor, dispersible tablets or powder for oral suspension administered orally once daily (QD) at a weight-based dose, and followed by the treating physicians at the participating sites. Participants will receive standard of care treatment and procedures for management of SCD, including an initial visit and routine visits at least every 12 weeks (± 7 days) for clinical and laboratory assessments per standard of care and re-supply of voxelotor. A safety follow-up visit will be conducted 28 days (± 7 days) after the last dose of investigational product.~This EAP may continue until such time that voxelotor is commercially available for patients aged 6 months to 11 years, or the Sponsor discontinues the voxelotor EAP.
The intent of this open-label, multicenter Expanded Access Program (EAP) is to provide early access to treatment with voxelotor prior to market authorization for pediatric patients age 6 months to 11 years with sickle cell disease (SCD) who have no alternative treatment options and are ineligible to participate in clinical trials of voxelotor. Cohort A, ages 4 to 11 years is now closed to enrollment.