Bluebird Bio Inc (NASDAQ:BLUE) has signed an agreement with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency for fast-track approvals on ongoing clinical trials. The company is working on developing a gene therapy for a rare blood disorder called beta-thalassemia, and the deal will help in achieving strategies for speedier regulatory approvals. LentiGlobin will help treat the blood disorder that is prevalent in around 15,000 people in the U.S. and Europe and over 300,000 globally.
Under the deal, apart from the existing trials that Bluebird is conducting for the gene therapy, the company is required to undergo two more studies. They have to divide the study participants into adult and children category and observe these patients for two years. If the gene therapy can avoid the blood transfusions requirement for a year, it would be deemed successful by the regulatory bodies.
Bluebird Pleased With Outcome
In an official statement, the company’s Chief Medical Officer David Davidson said that they were contented with the results of the communication with the regulatory bodies. The company is positive that this would spearhead their vision of a gene therapy to help the patients fight beta-thalassemia in a better manner.
The company is now working on completing the trials and getting the approvals for becoming a pioneer in the therapy in the European and the U.S. markets. The LentoGlobin BB305 treatment is aimed at countering the ability of the blood disorder to slow down the production of hemoglobin in the body. If the clinical trials meet the desired objectives, then just injecting the drug once can help in correcting the genetic disorder. The treatment will also help the patients in avoiding anemia.
Regulatory Trials To Start Soon
The company is mulling to get on board 30 patients who will be divided equally for two trials to be conducted for two years. On the basis of the results of the studies conducted so far, Bluebird Bio Inc (NASDAQ:BLUE) is optimistic of a positive outcome and securing regulatory approvals. The treatment has already been designated as a breakthrough therapy by FDA, and this is a bonus for the company. The road ahead seems to be excellent for the company to become a pioneer in this field.
