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MHRA grants bluebird bio’s LentiGlobin gene therapy an ‘innovation passport’

The Innovative Licensing and Access Pathway scheme aims to help speed approval of innovative treatments

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted bluebird bio’s potential sickle cell disease gene therapy a new ‘innovation passport’.

The Innovative Licensing and Access Pathway scheme was launched in January to accelerate the regulatory approval and reimbursement of innovative treatments.

LentiGlobin, a gene therapy for sickle cell disease, has been granted access to the scheme by the Innovative Licensing and Access Pathway steering group, comprised of the MHRA, National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC).

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

"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,” said John James, chief executive of the Sickle Cell Society.

“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,” he added.

LentiGlobin is currently being investigated as a potential one-off treatment for sickle cell disease in the ongoing phase 1/2 HGB-206 and the phase 3 HGB-210 studies.

These studies were relaunched earlier this month after the US Food and Drug Administration (FDA) lifted a clinical hold on the LentiGlobin sickle cell studies.

The clinical holds were initially placed on the trials after bluebird bio said that it had received reports of a suspected unexpected serious adverse reaction (SUSAR) of myelodysplastic syndrome (MDS) in a patient from group C of the phase 1/2 HGB-206 study in February.

At the same time, bluebird bio said that another SUSAR of acute myeloid leukaemia (AML) had been reported in the HGB-206 study.

bluebird bio later announced that after further assessment following the review of results from additional tests of the MDS case, the treating investigator has concluded the patient did not have MDS and revised the diagnosis instead to transfusion-dependent anaemia.

bluebird bio also reported in March that it is ‘very unlikely’ that the SUSAR of AML was related to the BB305 lentiviral vector (LVV) used for LentiGlobin gene therapy.

“With this [innovation passport] 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 lead, Northern Europe Cluster 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.

Article by
Lucy Parsons

30th June 2021

From: Regulatory



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