FDA approves first gene therapies to treat patients with sickle cell disease

The US Food and Drug Administration (FDA) on Friday approved two milestone treatments, Casgevy (exagamglogene autotemcel [exa-cel]), and Lyfgenia (lovotibeglogene autotemcel [lovo-cel)], representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD).

In what was seen as a land-mark decision, the FDA granted approval of Casgevy to Vertex Pharmaceuticals (Nasdaq: VRTX) and of Lyfgenia to bluebird bio (Nasdaq: BLUE).

Additionally, the agency pointed out, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.