Sickle cell gene therapy granted MHRA ‘innovation passport’

Sickle cell gene therapy granted new ‘innovation passport’ under MHRA scheme

bluebird bio’s promising new treatment for the rare blood disorder, sickle cell disease (SCD), has been awarded an ‘Innovation Passport’ under a new UK approval process to bring innovative medicines more rapidly to patients.

LentiGlobin™ for Sickle Cell Disease (bb1111), has been awarded an ‘Innovation Passport’ by the Innovative Licensing and Access Pathway Steering Group, consisting of the Medicines and Healthcare products Regulatory Agency (MHRA), National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC).

Launched in January this year, the passport aims to accelerate the treatment through regulatory approval and reimbursement, in a scheme known as the Innovative Licensing and Access Pathway (ILAP).[1]

The move may mean people with sickle cell can benefit from gene therapy much sooner than through standard, separate regulatory and reimbursement pathways.[1] If approved, bluebird bio‘s LentiGlobin could become the first one-time, potentially curative treatment for the rare blood disorder in the UK.

John James, Chief Executive of the Sickle Cell Society, said: “We’re thrilled to hear that another potential treatment option for sickle cell disease is on the horizon and wholeheartedly welcome the Innovative Licensing and Access Pathway Steering Group’s efforts to accelerate regulatory approvals and reimbursement. Sickle cell is a chronic, life-long condition characterised by unpredictable pain crises which can require hospitalisation and have a significant detrimental impact on an individual’s health and quality of life. That’s why it’s so essential that we continue to focus on developing new tools and treatment options that enable patients to have more pain free days and a better quality of life.”

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

Reference

[1] Gov.uk. Innovative Licensing and Access Pathway. 2021. Available at: https://www.gov.uk/guidance/innovative-licensing-and-access-pathway. Accessed 24 June 2021.