Bluebird bio, a leading American biotech has been pioneering range of gene therapies for rare diseases such as severe sickle cell disease and neurological disorder adrenoleukodystrophy, as well as treatments for multiple myeloma, a plasma cell cancer.
Although the technology is still undergoing clinical trials, the company is eyeing a launch in Europe before its home market and is therefore, in talks with UK medicine regulators about the potential launch.
Bluebird’s Richard Morgan said: “Europe will be the first market. We’re having very pro-active early discussions. We don’t plan to come and surprise governments.
“The EMA (European Medicines Agency) has attractive adaptive pathways and works very closely with companies coming forward with new methodologies.”
The ongoing discussions are with the UK’s commercial medicines regulator NICE and its EU counterparts about the move.
Attributable to GSK’s recent decision to put its own division in the field up for sale as part of a wider £1bn cost-cutting drive, rare diseases have been thrown into the spotlight. Rare diseases are defined as affecting fewer than one in 2,000 people.
Bluebird has a market value of over $4bn (£3bn) on New York’s Nasdaq stock exchange and specialises in bone marrow transplants to cure patients
with faulty immune systems.
