bluebird bio’s application for its sickle cell disease (SCD) gene therapy has been accepted by the US Food and Drug Administration (FDA) for priority review.
The company is specifically seeking approval for lovotibeglogene autotemcel (lovo-cel) to treat SCD patients aged 12 years and older who have a history of painful complications associated with the disease.
Affecting approximately 100,000 people in the US, SCD is a life-long, incurable genetic disease that causes red blood cells to take a distinct crescent shape, which can block blood vessels and affect the way oxygen is carried around the body.
The disease can cause serious health problems including anaemia, fatigue, episodes of pain and chronic end-organ damage.
Andrew Obenshain, bluebird’s chief executive officer, said: “The burden that people living with SCD and their families live with today is staggering. Beyond extreme pain crises that send patients to the hospital, SCD progression is associated with grave long-term consequences.”
bluebird’s lovo-cel is an investigational one-time treatment designed to add functional copies of a modified form of the beta-globin gene into a patient’s own haematopoietic stem cells.
Following treatment, a patient’s red blood cells can produce anti-sickling haemoglobin that decreases the proportion of sickle haemoglobin, with the goal of reducing sickled red blood cells, haemolysis and other complications.
The company’s US submission is backed by efficacy results from 36 patients with 32 months of follow-up data and two patients with 18 months of follow-up. Safety data from 50 patients, including six patients with six or more years of follow-up is also included in the application.
Obenshain said: “The FDA’s acceptance of our Biologics License Application for lovo-cel moves us one step closer in bringing a potentially transformative therapy to the SCD community that is long overdue… We look forward to working with the agency on its review.”
If approved, lovo-cel will be bluebird’s third ex-vivo gene therapy approved in the US for a rare genetic disease, and its second FDA approval for an inherited haemoglobin disorder.
In September last year, the company’s Skysona (elivaldogene autotemcel) gene therapy was granted FDA accelerated approval to slow the progression of neurologic dysfunction in certain patients with cerebral adrenoleukodystrophy, and its Zynteglo (betibeglogene autotemcel) gene therapy was approved by the regulator the prior month to treat beta-thalassaemia.