bluebird bio’s sickle cell gene therapy granted MHRA ‘innovation passport’

bluebird bio’s gene therapy for sickle cell disease LentiGlobin has been granted an ‘innovation passport’ from the UK Medicines and Healthcare products Regulatory Agency (MHRA).

The ‘innovation passport’ pathway, launched in January, offers a new UK approval process – one which aims to reduce the time to market for innovative medicines.

LentiGlobin has been awarded the designation by the Innovative Licensing and Access Pathway Steering Group, which consists of the MHRA, National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC).

In a statement, bluebird said that if LentiGlobin is approved it could become the first one-time treatment for the rare blood disorder in the UK.

LentiGlobin for sickle cell disease is being studied in a broad clinical development programme, which includes the completed Phase I/II HGB-205 study, the ongoing Phase I/II HGB-206 study and the ongoing Phase III HGB-210 study.

“With this designation, the UK progresses another important step towards making gene therapy available for people with sickle cell disease,” said Nicola Redfern, VP general manager UK and Northern Europe Cluster lead of bluebird bio.

“The designation clearly shows that the MHRA understands the value and enormous potential of innovative gene therapy for people with rare and serious inherited disorders such as haemoglobinopathies,” she added.

Sickle cell disease is a rare inherited genetic disorder of the haemoglobin – a protein found in red blood cells that is responsible for the colour of the cell and for carrying oxygen around the body. In the UK, the rare genetic blood disorder affects approximately 15,000 people.