Bluebird cuts deal with regulators, plots path to first-ever US gene therapy approval
- Bluebird Bio, the closely-watched biotechnology and gene therapy firm, has struck a deal with U.S. and European regulators to map out an accelerated approval pathway for its gene therapy LentiGlobin for the treatment of beta-thalassemia, a devastating blood disorder that reduces the production of hemoglobin (there are an estimated 15,000 patients in the U.S. and Europe and nearly 300,000 worldwide)
- Under the agreement with the FDA and EMA, Bluebird will have to run two additional trials (beyond the promising but miniscule four-person trial it has already run and two other trials underway with sickle cell and beta-thalassemia patients) that will each be formed as 15-patient open-label studies (rather than randomized placebo trials). The trials will be separated into adults/adolescents and children-comprised arms, and Bluebird will have to monitor the patients for two years. To be a success, the therapy will have to eliminate the need for patients' blood transfusions for one year.
- Bluebird would use the data from the various existing and new trials for a regulatory filing in the U.S. and Europe. If it succeeds, LentiGlobin would be the first U.S.-approved gene therapy and the second Europe-approved gene therapy other than UniQure's Glybera (unless Glaxo wins approval for its treatment for a subset of children with ADA Severe Combined Immune Deficiency who cannot find suitable marrow donors).
Bluebird's trials will be among the most closely-watched studies in the entire biotechnology industry over the next several years. Gene therapies, including many that appeared promising at first, have been heartbreakers for the industry on multiple occasions.
The FDA and the EMA's willingness to play ball with Bluebird also underscores a growing trend of regulators trying to overcome bureaucratic hurdles to get potentially promising therapies to the market faster.
"This feedback brings us closer to achieving our vision of delivering one-time, potentially transformative gene therapy to patients," said Bluebird chief medical officer David Davidson in a statement.