FDA Approved Voxelotor for Sickle Cell Disease

The medication also has fast-track, orphan drug, and uncommon pediatric disease status. The FDA has approved a breakthrough treatment designation for voxelotor (previously called GBT440, Global Blood Therapeutics) for the treatment of sickle cell disease (SCD).

Voxelotor is being developed as a disease-modifying treatment for SCD. The breakthrough treatment designation for voxelotor depended on clinical information submitted from the following studies:

  • Preliminary safety and efficacy information from part A of the phase 3 HOPE study (GBT440-031)
  • Phase 1/2 study and open-label extension in adults (GBT440-001/024)
  • Ongoing phase 2 HOPE-KIDS 1 study in children age 6 to 17 (GBT440-007)
  • Compassionate Access experience in adults with severe SCD (not eligible for the HOPE study)

Voxelotor is being developed as an oral, once-daily treatment for patients with SCD. Voxelotor works by increasing hemoglobin’s affinity for oxygen. The developer accepts voxelotor blocks polymerization and the resultant sickling of red blood cells because oxygenated sickle hemoglobin does not polymerize.

With the potential to restore normal hemoglobin function and improve oxygen delivery, the company believes that voxelotor may potentially alter the course of SCD.

The FDA has approved voxelotor fast-track, orphan drug, and uncommon pediatric disease designations for the treatment of patients with SCD, in acknowledgment of the critical requirement for new SCD medications.

In the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization) study Voxelotor is at present being assessed, a phase 3 clinical examination in patients 12 years old and older with SCD.

Also, in the ongoing phase 2a HOPE-KIDS 1 study voxelotor is being studied, an open-label, single-and multiple-dose study in pediatric patients (ages 6 to 17 years) with SCD. HOPE-KIDS 1 is assessing the safety, tolerability, pharmacokinetics, and exploratory treatment effect of voxelotor.


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