Sickle cell therapy granted innovation passport by MHRA

pharmafile | June 30, 2021 | News story | Research and Development MHRA, SCD, brexit 

Bluebird bio has been granted an innovation passport, under the new UK approval process, for LentiGlobin (bb1111) a promising treatment for the rare blood disorder, sickle cell disease (SCD).

LentiGlobin is an investigational gene therapy treatment currently being investigated in Phase I, II, and III trials.

The innovation passport has been awarded by the Innovative Licensing and Access Pathway (ILAP) Steering Group, consisting of the MHRA, NICE, and the Scottish Medicines Consortium (SMC). The passport was launched in January this year, following the UK’s exit from the EU, and aims to accelerate the treatment through regulatory approval and reimbursement.

Nicola Redfern, VP and General Manager of the UK and Northern Europe Cluster, and 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.”

SCD is a rare blood disorder of the haemoglobin, the protein in red blood cells that is responsible for the colour of the cell and for carrying oxygen around the body.  The inherited genetic disorder affects around 15,000 people in the UK.  The main symptoms are anaemia and episodes of severe pain, caused by affected blood cells sticking together and blocking blood vessels – known as a ‘sickle cell crisis’.

Over time people with sickle cell can experience damage to organs such as the liver, kidney, lungs, heart and spleen. As a result, the condition may also shorten life-expectancy. In the UK, people with sickle cell disease have a mean life expectancy of 60 years – compared to a national average of 78.7 – 82.7 years.  Currently, the only curative treatment is a bone marrow transplant; however, this is only suitable for a very limited number of individuals who have a matched donor.

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.”

Kat Jenkins

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