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The approval of bluebird bio’s LYFGENIA gene therapy for patients ages 12 and older with sickle cell disease and a history of vaso-occlusive events was deemed a monumental development yet concerns over high pricing and safety concerns stunt the commercial launch of the gene therapy.
bluebird bio, Inc. announced that the FDA has granted approval for LYFGENIA™ (lovotibeglogene autotemcel), a one-time gene therapy for the treatment of sickle cell disease in patients aged 12 and older with a history of vaso-occlusive events (VOEs). Also known as lovo-cel, LYFGENIA is designed to address the underlying cause of sickle cell disease.
Andrew Obenshain, CEO of bluebird bio, expressed excitement, stating:
“Bringing LYFGENIA to people living with sickle cell disease is a milestone that bluebird has been working toward for almost a decade.”
The therapy, a result of extensive collaboration and transparency, marks bluebird’s third FDA-approved ex vivo gene therapy for a rare genetic disease.
Regina Hartfield, President, and CEO of the Sickle Cell Disease Association of America Inc., stated:
“We’re enthusiastic…about the FDA’s approval of this therapy, which could change the lives of people and families affected by sickle cell disease.”
LYFGENIA works by adding a functional β-globin gene to patients’ own blood stem cells, leading to durable production of adult hemoglobin with anti-sickling properties (HbAT87Q). HbAT87Q has a similar oxygen-binding affinity to wild-type HbA and has the potential to reduce vaso-occlusive events (VOEs). The therapy is expected to be available at bluebird bio’s Qualified Treatment Centers in early 2024.
The FDA approval is based on data from the Phase I/II HGB-206 study, with 94% of patients experiencing the resolution of severe VOEs. Common adverse reactions include stomatitis, thrombocytopenia, and neutropenia. However, there warrants a long-term safety and efficacy follow-up study will monitor patients for 15 years as LYFGENIA has a boxed warning highlighting hematologic malignancy.
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During LYFGENIA’s clinical trial, two cases of acute myeloid leukemia were reported after treatment. However, it was determined that the cases were unlikely to be related to viral vector insertion from the gene therapy. bluebird has noted that the commercial LYFGENIA is now produced using a different process. Long-term risks and potential effects, including infertility, are still under evaluation. The safety concerns and high pricing of LYFGENIA (from $3.1 million) have caused a roadblock for the company with stock prices dropping by ~40%.
In comparison to bluebird bios’ rival therapy Vertex’s CASGEVY which also received approval from the FDA, did not come with a boxed warning to date of hematologic malignancy.
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