This section highlights FDA-related milestones and regulatory updates for drugs developed by bluebird bio (BLUE).
Over the past two years, bluebird bio has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Betibeglogene, Lovotibeglogene, and LYFGENIA. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Betibeglogene autotemcel (beti-cel) - FDA Regulatory Timeline and Events
Betibeglogene autotemcel (beti-cel) is a drug developed by bluebird bio for the following indication: Gene therapy in adult, adolescent and pediatric patients with transfusion-dependent β-thalassemia (TDT).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Betibeglogene autotemcel (beti-cel)
- Announced Date:
- December 7, 2024
- Indication:
- Gene therapy in adult, adolescent and pediatric patients with transfusion-dependent β-thalassemia (TDT)
Announcement
bluebird bio, Inc announced updated data from patients with beta-thalassemia who require regular blood transfusions treated with betibeglogene autotemcel (beti-cel, approved commercially as ZYNTEGLO™) in clinical studies.
AI Summary
Bluebird bio, Inc. announced updated long-term data from clinical studies of betibeglogene autotemcel (beti-cel, known commercially as ZYNTEGLO™) in patients with beta-thalassemia who require regular blood transfusions. The data includes results from 63 adult and pediatric patients, with two of the earliest treated patients showing sustained treatment effects for over 10 years, and 81% of participants having more than five years of follow-up. The findings reveal that patients achieved durable transfusion independence along with normal or near-normal hemoglobin levels, regardless of age or genotype. These long-term outcomes also demonstrated effective control of iron levels, reducing the need for iron chelation therapy, which is important for managing iron overload. Overall, the updated data supports the lasting benefits and favorable safety profile of beti-cel in transforming the treatment options for individuals with severe beta-thalassemia.
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Lovotibeglogene autotemcel (lovo-cel; formerly LentiGlobin) - FDA Regulatory Timeline and Events
Lovotibeglogene autotemcel (lovo-cel; formerly LentiGlobin) is a drug developed by bluebird bio for the following indication: Sickle Cell Gene Therapy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Lovotibeglogene autotemcel (lovo-cel; formerly LentiGlobin)
- Announced Date:
- May 6, 2024
- Indication:
- Sickle Cell Gene Therapy
Announcement
bluebird bio, Inc announced that it has completed the first commercial cell collection for LYFGENIA (lovotibeglogene autotemcel), a one-time gene therapy for the treatment of sickle cell disease in patients with a history of vaso-occlusive events
AI Summary
bluebird bio, Inc. announced a major milestone by completing the first commercial cell collection for LYFGENIA (lovotibeglogene autotemcel), a new one-time gene therapy for sickle cell disease. This therapy is designed for patients who have experienced vaso-occlusive events, a common and painful complication of the disease. During the procedure, patients’ own cells are collected at Children’s National Hospital in Washington, DC, which is one of more than 60 Qualified Treatment Centers in bluebird’s network. LYFGENIA works by adding a functional β-globin gene to the patient’s blood stem cells, helping to produce adult hemoglobin with anti-sickling properties. bluebird bio’s accomplishment marks an important step toward offering a potentially life-changing treatment, moving gene therapy from clinical trials to real-world application and bringing hope to many patients and families affected by sickle cell disease.
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LYFGENIA - FDA Regulatory Timeline and Events
LYFGENIA is a drug developed by bluebird bio for the following indication: For patients with sickle cell disease who have a history of vaso-occlusive events (VOEs).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- LYFGENIA
- Announced Date:
- December 8, 2024
- Indication:
- For patients with sickle cell disease who have a history of vaso-occlusive events (VOEs).
Announcement
bluebird bio, Inc announced new and updated data from LYFGENIA™ (lovotobegligene autotemcel, or lovo-cel) gene therapy for patients with sickle cell disease who have a history of vaso-occlusive events (VOEs).
AI Summary
bluebird bio, Inc. announced new and updated data from its LYFGENIA™ (lovotobegligene autotemcel, or lovo-cel) gene therapy for patients with sickle cell disease and a history of vaso-occlusive events (VOEs). The study shows that patients maintained stable production of anti-sickling adult hemoglobin after treatment, with nearly all evaluable patients experiencing elimination or significant reduction in VOEs. Specifically, about 95% of patients achieved complete resolution of severe VOEs within 6 to 18 months post infusion, and these responses have been sustained over a median follow-up of more than three and a half years. Additionally, data from a focused analysis on patients with a history of overt or silent stroke demonstrated no stroke recurrence through up to nine years of follow-up. These findings suggest that LYFGENIA may offer durable, transformative benefits for people living with sickle cell disease.
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