bluebird bio, Inc. (Nasdaq: BLUE) (“bluebird bio” or “bluebird”) today announced the U.S. Food and Drug Administration (FDA) has approved LYFGENIA™ (pronounced as ‘lif-JEN-ee-uh’) (lovotibeglogene autotemcel), also known as lovo-cel, for the treatment of sickle cell disease in patients ages 12 and older who have a history of vaso-occlusive events (VOEs). LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.
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, chief executive officer, bluebird bio. “LYFGENIA has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating vaso-occlusive events. 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.”
“We’re enthusiastic at the Sickle Cell Disease Association of America Inc. about the FDA’s approval of this therapy, which could change the lives of people and families affected by sickle cell disease,” said Regina Hartfield, president and CEO of the Sickle Cell Disease Association of America Inc. “As the national advocacy organization for people with sickle cell, we’re strongly supportive of the new potentially curative option for treatment and excited for the future.”
“People living with sickle cell disease face potentially devastating health consequences, diminished quality of life, and harmful stigma as a result of their disease,” said Julie Kanter, M.D., a LYFGENIA investigator and director of the University of Alabama Birmingham Adult Sickle Cell Clinic and associate professor in the Division of Hematology and Oncology. “Today we can celebrate the availability of a potentially transformative new therapeutic option made possible by the incredible courage of patients and families who participated in clinical studies.”
“Sickle cell disease was the first genetic disorder to be characterized at the molecular level a half-century ago and today – after decades of waiting – we finally have a therapy that addresses the underlying cause of this devastating disease,” said John Tisdale, MD, Chief, Cellular and Molecular Therapeutics Branch at the National Heart, Lung, and Blood Institute (NHLBI), who served as principal investigator on the HGB-206 study and was a key collaborator throughout the clinical development program. “The development of this therapy has been marked by transparency and collaboration that laid the groundwork for other technologies to follow, and the NIH is extremely pleased to have supported this advance.” NHLBI is part of the National Institutes of Health.
Sickle cell disease is a complex and progressive genetic disease associated with unpredictable and debilitating vaso-occlusive events (VOEs).1,2,3,4 In sickle cell disease, high concentrations of sickle hemoglobin (HbS) in red blood cells (RBCs) cause them to become misshapen, sticky and rigid, with a shorter life span, which manifests acutely as hemolytic anemia, vasculopathy and vaso-occlusion. The burden associated with VOEs is pervasive and can affect every aspect of life for patients and their families and caregivers.
LYFGENIA works by permanently adding a functional β-globin gene to patients’ own hematopoietic (blood) stem cells (HSCs). Durable production of adult hemoglobin with anti-sickling properties (HbAT87Q) is expected following successful engraftment. HbAT87Q has a similar oxygen-binding affinity to wild-type HbA, limits sickling of red blood cells, and has the potential to reduce vaso-occlusive events (VOEs).
LYFGENIA will be available at bluebird bio’s established network of Qualified Treatment Centers (QTCs), which receive specialized training to administer complex gene therapies like LYFGENIA. Information on bluebird’s QTC network, as well as personalized support focused on the needs of each patient throughout their treatment journey, is available through bluebird’s patient support program, my bluebird support. Patients can call 833-888-NEST (833-888-6378) or visit mybluebirdsupport.com for more information. bluebird plans to make the therapy available in early 2024.
LYFGENIA was granted Priority Review in June 2023. The Company did not receive a Rare Pediatric Disease Priority Review Voucher as part of the review. LYFGENIA was previously granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation.
