Summary
- bluebird bio gains regulatory approval in Europe for its gene therapy Zynteglo to treat patients with beta-thalassemia, which was the very first approval for it.
- A majority of those with beta-thalassemia, who were treated with Zynteglo, were shown to achieve transfusion independence of 12 months or more.
- bluebird bio has multiple catalysts on the way, which include NDA filing of Zynteglo to the FDA by the end of 2019 and potentially U.S. marketing approval in 2020.
- bluebird bio has $1.73 billion in cash as of March 31, 2019, which is expected to fund operations into 2022.
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bluebird bio (BLUE) announced that it had received European approval for its gene therapy Zynteglo to treat patients with beta-thalassemia. This was the first approval for the biotech which was an important milestone to reach. However, this is only the beginning. That's because bluebird bio expects to possibly file for approval of Zynteglo to the FDA by the end of this year. That would set up a potential U.S. approval for the gene therapy in 2020 for the very same indication.
