“CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death. The acceptance of the MAA for eli-cel is a critical milestone in our continued collaboration with the EMA to potentially deliver an autologous gene therapy for boys with CALD,” said
Data from the Phase 2/3 Starbeam study (ALD-102) formed the basis of the MAA, which is also supported with data from the ongoing Phase 3 ALD-104 study and the long-term follow-up study (LTF-304). The most recent results from these studies were presented at the 46th Annual Meeting of the
Eli-cel is a one-time investigational gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic (blood) stem cells (HSCs) that have been transduced ex vivo with the Lenti-D lentiviral vector (LVV). The addition of a functional gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to allow for the breakdown of very-long-chain fatty acids (VLCFAs) that accumulate to toxic levels in the brain. There is no need for donor HSCs from another person.
The EMA accepted eli-cel gene therapy for the treatment of CALD into its Priorities Medicines scheme (PRIME) in
Eli-cel is not approved for any indication in any geography.
bluebird bio is currently enrolling patients for a Phase 3 study (ALD-104) designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Contact firstname.lastname@example.org for more information and a list of study sites.
Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with eli-cel for CALD and completed two years of follow-up in bluebird bio-sponsored studies.
The Phase 2/3 Starbeam study (ALD-102) has completed enrollment.
About Cerebral Adrenoleukodystrophy (CALD)
Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that is estimated to affect one in 21,000 male newborns worldwide. ALD is caused by mutations in the ABCD1 gene that affect the production of adrenoleukodystrophy protein (ALDP) and subsequently cause toxic accumulation of very long-chain fatty acids (VLCFAs) primarily in the adrenal cortex and white matter of the brain and spinal cord.
Approximately 40% of boys with adrenoleukodystrophy will develop CALD, the most severe form of ALD. CALD is a progressive neurodegenerative disease that involves breakdown of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms of CALD usually occur in early childhood and progress rapidly, if untreated, leading to severe loss of neurologic function, and eventual death, in most patients. CALD is associated with six major functional disabilities (MFDs), which severely compromise a patient’s ability to function independently: loss of communication, cortical blindness, need for tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement.
Although allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been shown to have a beneficial effect on clinical indices of disease and long-term survival and can arrest disease progression if performed at the early stage of cerebral involvement, it has significant associated risks, such as transplant-related mortality (TRM), graft failure or rejection, graft-versus-host disease, and potential for opportunistic infections. Safety outcomes are typically more favorable if allo-HSCT is performed using cells from a human leukocyte antigen (HLA)-matched sibling donor.
Early diagnosis of CALD is important, as the outcome of available treatment varies with the clinical stage of the disease. Newborn screening for ALD is a critical enabler of early diagnosis and thus of successful treatment of ALD. Once a patient has been diagnosed with ALD, regular MRI scans are critical to detect white matter changes indicative of progression to CALD.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders, including cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in
eli-cel and bluebird bio are trademarks of bluebird bio, Inc.
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the company’s expectations and plans for regulatory approval for and commercialization of eli-cel in the
Source: bluebird bio, Inc.