FDA Advisory Group Permits Bluebird Gene Therapy Amid Risks

Bluebird bio has persuaded an expert committee that the benefits of its gene therapy possibility, eli-cel, exceed the risks, at least in certain patients, after encountering major safety concerns from FDA reviewers. The FDA invited its Cellular, Tissue, and Gene Therapies Advisory Panel to hear appeals and consider the risks and benefits of elivaldogene autotemcel on the first day of a high-stakes, two-day conference focused on two of the company’s gene therapy applications. Bluebird biogene therapy is being evaluated for the treatment of patients under the age of 18 who have cerebral adrenoleukodystrophy (CALD).

The panel ruled 15-0 that the advantages of eli-cel exceed the dangers for specific groups of CALD patients after hearing hours of data on the drug’s safety and effectiveness. Several judges agreed that the medicine is appropriate for individuals who do not have a stem cell donor match. For individuals who have a donor, this “could be left up to the physician and patient’s decision, according to Donna Roberts, M.D., a professor in the Medical University of South Carolina’s department of radiology and radiological sciences.

Overall, she believes this is a critical product to have in the marketplace.

Despite hearing extensive safety warnings from FDA personnel, the panel made its decision. The danger of myelodysplastic syndrome (MDS), a kind of malignancy that developed in three of 67 individuals (4%) treated with the medication, was highlighted in FDA briefing papers prior to the meeting.

At the conference, Leah Crisafi, M.D., a clinical investigator with the FDA’s Office of Tissues and Advanced Therapies, stated that eli-cel treatment has a high risk of malignancy. She observed that the present rate of malignancy is 4% but that it is expected to rise since the length of follow-up during the drug’s drug trials was very limited and may have been inadequate for malignancy to arise.

Nonetheless, the committee’s approval puts the medicine on firm footing ahead of the FDA’s September 16 decision date. The agency does not always follow the recommendations of its advisory committee, although it usually does. The medicine was approved in Europe last summer under the trade name Skysona.

The vote gives Bluebird a much-needed victory following a string of losses in recent years. After receiving European approval in June 2019 for betibeglogene autotemcel to treat beta thalassemia, the company was forced to leave the market last year after failing to persuade funders of the therapy’s benefits. After its marketing ambitions in Europe were crushed and regulatory delays lurked in the United States, the company downsized 30% of its workforce in April.

The FDA advisory committee examination of the company’s beti-cel product has already been scheduled.