EMA may approve new gene therapy product by Bluebird Bio Company

European Medicines Agency is considering the application of the American Bluebird Bio for the registration of cerebral adrenoleukodystrophy drug.

It is assumed that the American company Bluebird Bio is now several months away from the approval of its gene therapy product for a rare disease - cerebral adrenoleukodystrophy. In the EU - EMA has begun a rapid process of reviewing the registration dossier for a drug, known as eli-cel. CThis drug has been created on the basis of the patient's autologous stem cells and a genetically modified virus, which delivers functional copies of the mutant gene responsible for the development of the disease to the cell nuclei. The manufacturer says that the drug could change the prospects for people diagnosed with cerebral adrenoleukodystrophy, which is a severe neurodegenerative disease.
A couple of weeks ago, the company released new data from STARBEAM phase 2/3 trial, which showed that 87% of patients with this diagnosis are still alive and have no significant functional impairment after a single administration of eli-cel for about two years.


SOURCE: PHARMA PHORUM