The U.S. Food and Drug Administration (FDA) has expanded the age range for the use of CRISPR therapy intended to treat sickle cell anemia and beta-thalassemia. This treatment is now available for children starting from the age of two, whereas previously it was only used from age 12.
Vertex Pharmaceuticals reported that the drug exagamglogene autotemcel (Casgevy) is approved for treating transfusion-dependent beta-thalassemia, as well as sickle cell disease accompanied by recurrent vaso-occlusive crises. The mechanism of action of the drug involves turning off the BCL11A gene. This gene usually suppresses fetal hemoglobin production after birth, but when it is turned off, fetal hemoglobin begins to replace the defective one, ensuring necessary oxygen delivery.
The FDA's decision was based on preliminary results obtained during ongoing open, non-randomized Phase III trials, namely CLIMB SCD-151 and CLIMB THAL-141. After a single course of exagamglogene autotemcel, the hemoglobin level in the subjects normalized. In patients with sickle cell anemia, episodes of vaso-occlusive crises ceased, and in people with beta-thalassemia, the need for regular blood transfusions disappeared.
After receiving the license for the drug for the new indication, the manufacturer is required to conduct additional post-marketing prospective multicenter observational studies. These studies are necessary to more accurately determine the long-term safety of the therapy in this new patient group.
In another area, the editorial board of Nature Medicine withdrew an article that was published in February 2026. The authors of this work claimed that administering chemotherapy in the first half of the day could nearly double median survival rates in non-small cell lung cancer. However, shortly after publication, experts began noticing inconsistencies in the study. Among the identified problems were the absence of any dropout among the 210 participants due to side effects, which is extremely rare in oncology studies, as well as changes in the design and outcome measures during the study itself. Furthermore, the pharmacokinetics of the drug used do not suggest dependence on the time of administration. Moreover, if the conclusions drawn were correct, they would have revolutionized oncology, which did not actually happen.
