News

The U.S. Food and Drug Administration has granted orphan drug status to Kind Pharmaceuticals’ investigational oral small molecule AND017 to treat sickle cell disease (SCD). This designation is granted to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides incentives like…

Editas Medicine has announced proof-of-concept data for a new gene-editing approach for sickle cell disease (SCD) that does not involve a stem cell transplant. The strategy instead makes use of targeted lipid nanoparticles, or LNPs, which are tiny vesicles made of fatty molecules. They are used to deliver…

Patient enrollment has been completed for the Phase 3 RISE UP study, a clinical trial that’s assessing the safety and efficacy of the oral therapy mitapivat in people with sickle cell disease (SCD). More than 200 patients, 16 and older, have been enrolled, and results from the study…

Zydus Lifesciences is launching a proof-of-concept Phase 2a clinical trial to evaluate the safety and efficacy of its oral candidate desidustat in people with sickle cell disease (SCD). The study is the result of a collaboration with the Indian Council of Medical Research (ICMR). “Public-private partnerships in the…

Women with sickle cell disease (SCD) experience significant challenges with healthcare while struggling with both the physical and social toll of the disease, a new study from the University of Pennsylvania reports. Managing physical pain, facing emotional challenges, and feeling stigma from healthcare providers, especially when transitioning from…

Enlargement of the left atrium, one of the heart’s upper chambers, is associated with a greater sickle cell disease (SCD) burden for patients, with more emergency department visits and blood test abnormalities, according to a new study from the U.S. A larger left atrium is believed to be a…

Preschool-aged children with sickle cell disease (SCD) living in so-called food deserts, or areas where access to healthy food is limited, and those without reliable transportation are at a higher risk of experiencing health issues than other youngsters with SCD, even when they receive good medical care, a study…

Mitem Pharma has acquired worldwide rights to Desferal (deferoxamine), a therapy commonly used to lower excessive and damaging iron levels induced by blood transfusions in people with sickle cell disease (SCD) or other anemias. The acquisition was reached in an agreement between Mitem and Novartis, which…

The number of stroke cases among adults and children with sickle cell disease (SCD) continues to increase in at least one U.S. state, indicating a need for greater adherence to standard-of-care blood transfusions and more attention paid to factors that can be controlled to reduce the risk of…

Pfizer has voluntarily withdrawn Oxbryta (voxelotor), a conditionally approved oral therapy for sickle cell disease (SCD), from worldwide markets, saying clinical data shows the treatment’s benefit “no longer outweighs the risk.” Citing an imbalance in vaso-occlusive crises (VOCs) and deaths occurring in patients given Oxbryta in postmarketing…