Bluebird bio receives US FDA nod for sickle cell therapy

The US Food and Drug Administration (FDA) has approved bluebird’s Lyfgenia (lovotibeglogene autotemcel) for the treatment of sickle cell disease. 

Approved

 for patients aged 12 or older with a history of vaso-occlusive events (VOEs), Lyfgenia is a one-time gene therapy with the potential to resolve VOEs and is custom-designed to treat the underlying cause of the disease. The firm plans to make the therapy available by early 2024. 

“Bringing Lyfgenia to people living with sickle cell disease is a milestone that bluebird has been working toward for almost a decade—and one that members of the sickle cell disease community have been waiting on for much longer,” said Andrew Obenshain, CEO of bluebird bio.

Lyfgenia functions by integrating a functioning β-globin gene into the patients’ own hematopoietic stem cells (HSCs). After successful engraftment, sustained generation of adult hemoglobin with anti-sickling characteristics (HbAT87Q) is anticipated. This can potentially lessen VOEs, restrict red blood cell sickling, and has an oxygen-binding affinity comparable to that of wild-type HbA. 

“This therapy has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating VOEs. This approval also 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, cementing our position as a gene therapy leader,” said Obenshain. 

The sickle cell therapy, Lyfgenia, also known as lovo-cell will be available at the firm’s established network of qualified treatment centers (QTCs). As per the firm, these centers specialize in administering complex gene therapies. 

As the FDA’s decision date drew closer, Novartis purchased bluebird’s priority review voucher (PRV) worth $103 million for the sickle cell disease gene therapy treatment in 

November 2023.

 Lyfgenia was granted priority review in June 2023. At the time, bluebird bio said that “the potential sale of a PRV would provide an important source of non-dilutive capital for bluebird ahead of the anticipated launch of lovo-cel.”  

On the same day as Lyfgenia received FDA approval, the regulatory body also authorized Vertex’s CRISPR/Cas9 gene-edited therapy Casgevy (exagamglogene autotemcel [exa-cel]), which is a sickle cell disease (SCD) treatment.