USANew treatments and approvals
US approval of a sickle cell and thalassemia gene therapy extended to children over 2 years
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
On July 1, 2026, the US Food and Drug Administration (FDA) approved exagamglogene autotemcel for children over 2 years of age. The treatment was previously given to people aged 12 and older. The patient's own stem cells are edited with CRISPR and given back. None of the 8 children with sickle cell disease assessed in the study had a severe crisis for 12 months. In thalassemia, 8 of 9 children did not need a blood transfusion for 12 months.
What does it mean for patients and families?
Before treatment, a strong preparatory treatment that empties the bone marrow is needed. Its risks should be discussed with the physician. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Sickle cell disease, Thalassemia
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.