Gene Editing Therapy Wins Fast Track Designation for Sickle Cell Disease
GPH101 is an investigational gene-edited autologous hematopoietic stem cell therapy designed to correct the mutation in the beta-globin gene and potentially curing the disease.
Sickle Cell Disease
Systemic Corticosteroids Tied to Vaso-Occlusive Episodes in Sickle Cell
Association seen among children and adults with sickle cell disease; association lower for those exposed to hydroxyurea
FDA Extends Review Period for Gene Therapies for Beta-Thalassemia, Cerebral Adrenoleukodystrophy
The FDA extended the target dates for beti-cel and eli-cel to allow time to review additional clinical information for both applications.
Oxbryta Approval Expanded to Younger Patients With Sickle Cell Disease
The approval for both applications was based on data from the open-label phase 2a HOPE-KIDS1 study, which evaluated the efficacy and safety of Oxbryta 300mg dispersible tablet in 45 patients aged 4 to 11 years with sickle cell disease.
Educational Burdens for Children With Sickle Cell Disease in the US Vs Other Nations
Researchers sought to determine the educational burdens of children with SCD in the US compared with other nations.
Siklos Approval Expanded to Include Adults With Sickle Cell Anemia
The approval was based on data from the European Sickle Cell Disease Cohort study, which evaluated the efficacy and safety of Siklos in 1077 adults with sickle cell disease.
ASH Issues Guideline on the Use of HSCT for Sickle Cell Disease
The use of HSCT for sickle cell disease is evolving, and no randomized controlled trials were identified in the evidence review for this guideline
Voxelotor Gets Priority Review for Pediatric Sickle Cell Disease
The sNDA and NDA are supported by data from the open-label phase 2a HOPE-KIDS1 study.
Treatment at Infusion Center Beneficial in Sickle Cell Disease
Outcomes significantly better for treatment of vaso-occlusive crises at infusion center versus emergency department
Ferriprox Approved for Transfusional Iron Overload Due to Sickle Cell Disease
The Food and Drug Administration (FDA) has expanded the approval of Ferriprox® (deferiprone) tablets to include the treatment of transfusional iron overload due to sickle cell disease (SCD) or other anemias in patients 8 years of age and older. Additionally, the oral solution formulation of Ferriprox has been approved for use in patients 3 years…
