US-based emerging company Bluebird Bio has still not disclosed any plans to bring its gene therapies to Australia but has secured European approval for ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene).
The company announced the European Medicines Agency approved ZYNTEGLO as the first gene therapy to treat patients 12 years and older with transfusion-dependent β-thalassemia (TDT).
TDT is a debilitating disease characterised by severe anaemia and lifelong dependence on red blood cell transfusions that can lead to iron overload.
ZYNTEGLO is expected to launch in Germany in the second half of this year. Launches are planned for Italy, France and the UK in 2020, followed by wider expansion across the EU. ZYNTEGLO will be submitted to the FDA by the end of this year in advance of the planned US launch.
The company has not disclosed any plans to launch ZYNTEGLO in Australia or anywhere in the southern hemisphere.
It is one-time gene therapy and the company has capped the cost at US$2.1 million.
It has proposed a value-based pricing approach under which they would only charge if the treatment works. The company says it will gamble 80 per cent of the price on a performance-based payment model.
Bluebird Bio has a contract manufacturing site in Germany with nine treatment centres in Europe, including four in Italy and three in Germany.
The company has a current market capitalisation of A$10 billion.