bluebird wins FDA approval for sickle cell gene therapy 

Dec. 11, 2023

On Friday — a historic day for sickle cell disease — the FDA approved bluebird bio's one-time gene therapy, lovo-cel, for the treatment of SCD in patients ages 12 and older who have a history of vaso-occlusive events (VOEs). 

Branded Lyfgenia, the gene therapy that has the potential to resolve VOEs — acute complications associated with SCD — and is custom-designed to treat the underlying cause of sickle cell disease. The therapy works by permanently adding a functional β-globin gene to patients’ own hematopoietic (blood) stem cells.

The FDA nod came on the same day that the agency approved Vertex Pharmaceuticals and CRISPR Therapeutics' CRISPR/Cas9 genome-edited cell therapy, exa-cel (branded Casgevy), for SCD patients 12 years and older with recurrent vaso-occlusive crises.

The timing for the approval may not have worked in bluebird's favor, as shares for the company fell after Lyfgenia received a black-box warning and was announced at a higher price point than the Vertex/CRISPR treatment.

According to the FDA, blood cancer has occurred in patients treated with Lyfgenia, necessitating a black box warning with information regarding this risk. Bluebird said the treatment would come with a $3.1 million price tag; Casgevy will cost $2.2 million.

Lyfgenia's approval marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder.