Author Archives: Marta Figueiredo PhD

Regulators in Brazil have approved Ferriprox (deferiprone), Chiesi Global Rare Diseases’ iron-binding oral treatment, for blood transfusion-induced iron overload in people with sickle cell disease (SCD) or other anemias. Ferriprox’s use was also approved in combination with other iron-binding, or iron-chelating, treatments for people with thalassemia major, a severe…

The Sickle Cell Disease Foundation (SCDF) will use a $1 million grant to educate sickle cell disease (SCD) patients living in the western U.S. about the COVID-19 vaccine and assist them in accessing vaccination programs. The goal is to increase the number of SCD patients in western U.S.

Children with sickle cell disease (SCD) as young as age 4 could be prescribed Oxbryta (voxelotor) if the U.S. Food and Drug Administration approves a supplemental new drug application (sNDA) submitted by the therapy’s developers. Global Blood Therapeutics (GBT) is asking the FDA to expand the use of…

The University of California, San Francisco (UCSF) has been awarded $8.39 million to fund a Phase 1/2 clinical trial of CRISPR_SCD001, the first non-viral and CRISPR-Cas9-based gene editing therapy for sickle cell disease (SCD). Researchers will use the gene-editing technology to attempt to correct a genetic mutation in the blood…

A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.

Global Blood Therapeutics (GBT) is currently focused on expanding the use of Oxbryta (voxelotor) to children, ages 4–11, with sickle cell disease (SCD). But research into its use with younger children, as well as studies into other potential SCD therapies, are also underway. “Sickle cell disease patients of all ages worldwide…

In children with sickle cell disease (SCD), ages 4–11, weight-based doses of Oxbryta (voxelotor) result in similar rises in hemoglobin levels and reductions in red blood cell destruction (hemolysis) to those observed in older patients at the approved dose, six-month data from the ongoing Phase 2 HOPE-KIDS 1…

The Saudi Food and Drug Authority (SFDA), in Saudi Arabia, will review Emmaus Life Sciences’ application for the approval of its oral medication Endari (L-glutamine) for treating sickle cell disease (SCD). Given that the SFDA’s review process typically takes 12 to 18 months, a decision is not expected before…

Six months of treatment with Oxbryta (voxelotor) safely and effectively increased hemoglobin levels and reduced red blood cell destruction, or hemolysis, in children ages 4–11 with sickle cell disease (SCD), according to data from the ongoing Phase 2 HOPE-KIDS 1 trial. These findings are consistent with those…

The U.S. Food and Drug Administration (FDA) has expanded the label of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to include patients ages 3 and older who have blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. The therapy was first approved…