News

NICE is not planning to recommend Casgevy for public reimbursement

Authorities in the U.K. have preliminarily recommended that Casgevy (exagamglogene autotemcel) not be reimbursed for eligible patients with sickle cell disease (SCD) under its national public health program based on uncertainties about the gene therapy’s cost-effectiveness. The drafted decision from the National Institute for Health and Care…

Sickle cell children may not get preventive care: Study

While children with sickle cell anemia — the most common form of sickle cell disease — are vulnerable to serious infections and stroke, many don’t receive the antibiotics or annual ultrasounds that national guidelines suggest could keep them healthier, a study found. Only about 1 in 5 children took…

SCD and SCT tied to worse COVID-19 outcomes in pooled analysis

People with sickle cell disease (SCD) or sickle cell trait (SCT) face an increased risk of hospitalization and mortality following infection with SARS-CoV-2, the virus that causes COVID-19, compared with the general population, according to a pooled analysis of published studies. Researchers also noted that kidney complications after COVID-19…

Conditional approval of Adakveo for SCD now revoked in UK

Novartis is recalling its sickle cell disease (SCD) treatment Adakveo (crizanlizumab) in the U.K., after the nation’s drug regulatory agency revoked the conditional approval it had previously granted to the antibody-based therapy. The U.K. Medicines & Healthcare products Regulatory Agency (MHRA) made its decision based on Adakveo’s failure…