New Data Published on Voxelotor for Pediatric Sickle Cell Disease Treatment


Voxelotor is the first and only FDA-approved medication for children with sickle cell disease.

New Data Published on Voxelotor for Pediatric Sickle Cell Disease Treatment

Phase 2 data from Global Blood Therapeutic's HOPE-KIDS 1 trial found that voxelotor (Oxbryta) improves hemoglobin (Hb) levels and markers of hemolysis and has the potential to mitigate sickle cell disease-related complications based on its mechanism as a Sickle hemoglobin (HbS) polymerization inhibitor.

These data were recently published in Pediatric Blood & Cancer. The featured phase 2A open-label, single and multiple-dose study assessed the efficacy, safety, and pharmacokinetics of voxelotor in pediatric patients with sickle cell disease.

Voxelotor was the first and only medication for sickle cell disease in children approved by the US Food and Drug Administration (FDA) in December 2021. The sickle hemoglobin polymerization inhibitor is now recommended for patients aged 4 and older.

According to the data, a cohort of patients between the ages of 4-11 who received voxelotor for up to 48 weeks displayed improved hemoglobin levels along with markers of hemolysis. Related complications can also potentially be mitigated.

Inestigators found no new safety signals to be concerned about and the therapy was well tolerated in trial patients. The phase 2 research has been presented at multiple meetings including 2021 European Hematology Association Congress, and the 2021 49th Annual Sickle Cell Disease Association of America National Convention.

Related Videos
Elna Saah, MD: Unraveling the Current Landscape of Sickle Cell Disease | Image Credit: Twitter
Hematopoietic Stem Cell Transplantation Improves Pediatric SCD Outcomes | Image Credit: Scott Graham/Unsplash
How Gene and Cell Therapy Is Developing in Dermatology
Joyce Teng, MD, PhD, discusses how therapeutic advances in fields like epidermolysis bullosa should progress treatment discourse in other rare dermatoses.
The Prospect of Pz-cel in RDEB Treatment, with Peter Marinkovich, MD
Comparing New Therapies for Dystrophic Epidermolysis Bullosa
Reviewing 2023 with FDA Commissioner Robert M. Califf, MD
Dunia Hatabah, MD | Image Credit: HCPLive
Ricky Safer: What Clinicians Need to Know About PSC
Ryan T. Fischer, MD: Long-Term Odevixibat Benefit for Alagille Syndrome
© 2024 MJH Life Sciences

All rights reserved.