Teplizumab authorised by the MHRA
The first ever immunotherapy for type 1 diabetes has now been licensed for use in the UK.
The Medicines and Healthcare products Regulatory Agency (MHRA) has confirmed teplizumab, also known as Tzield, is safe and effective at delaying the development of type 1 diabetes for people in the early stages of the condition.
Sanofi has received authorisation for teplizumab – the world's first licensed immunotherapy for type 1 diabetes, tackling the root cause of the condition – the immune system attack on insulin-making beta cells in the pancreas – for the first time.
By slowing this attack, teplizumab can protect beta cells and delay the need for insulin treatment in people with early type 1 diabetes for around three years on average.
The drug has been approved for people aged eight years and over in the UK who have early stage type 1 diabetes, the symptomless phase where the immune system has begun its attack, but the body is still able to produce some insulin.
Following authorisation in the US, the UK is the first country in Europe to be granted a licence. Until now, available options have focused solely on managing the symptomatic disease.
Type 1 Diabetes is an autoimmune, complex, and lifelong condition – and it can happen at any age even without family history.
It is growing in prevalence in the UK, with approximately 400,000 people living with type 1 diabetes, of which around 32,000 people are aged 19 years or younger.
It is known that type 1 diabetes is not caused by diet or lifestyle, but the causes of type 1 diabetes are currently not fully understood.
Type 1 diabetes develops gradually in three stages over months or years. Long before symptoms appear, the immune system mistakenly attacks the insulin-producing cells in the pancreas.
By stage 3 most of the insulin-producing cells are destroyed and people become symptomatic.
From stage 3, people experience symptoms like increased thirst and weight loss; this is usually when they are diagnosed.
Once diagnosed, they face a daily regimen of multiple insulin injections or pump use, continuous glucose monitoring, strict dietary management, and the risk of acute complications, including diabetic ketoacidosis, a potentially life-threatening condition.
Beyond the immediate physical demands, type 1 diabetes carries a psychological toll, impacting mental health, social activities, and overall quality of life.
The long-term complications of the disease include kidney disease, nerve damage, blindness, and cardiovascular issues.
Teplizumab has been the subject of multiple clinical studies involving more than 1,000 subjects with more than 800 people receiving teplizumab.
The pivotal TN-10 study demonstrated the capability of teplizumab to preserve beta cell function and reduce the need for exogenous insulin usage in those newly diagnosed.
A single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease to Stage 3 T1D diagnosis by a median time of 50 months from randomisation in the trial group (n=44) and 25 months in the placebo group (n=32), for a difference of 25 months.
The primary endpoint in this study was the time from randomisation to development of Stage 3 type 1 diabetes diagnosis. The observed adverse events were lymphopenia, leukopenia, neutropenia, blood bicarbonate decreased and rash.
More serious, but rarer reactions included cytokine release syndrome, decreased haemoglobin, thrombocytopenia, liver enzyme and bilirubin elevations.
Parth Narendran, Professor of Diabetes Medicine, said:“Teplizumab essentially trains the immune system to stop attacking the beta cells in the pancreas, allowing the pancreas to produce insulin without interference.
“This can allow eligible people to live normal lives, delaying the need for insulin injections and the full weight of the disease's daily management by up to three years. It allows people to prepare for disease progression rather than facing an abrupt emergency presentation.”
Karen Addington MBE, Chief Executive of Breakthrough T1D UK, said: “After years of research supported by Breakthrough T1D, I am delighted by the MHRA’s authorisation of teplizumab.
“We now have an immunotherapy that can delay the development of symptomatic type 1 diabetes and may help families avoid life-threatening complications, like diabetic ketoacidosis (DKA).
“What teplizumab offers is time free from the daily challenges of type 1 diabetes management, a potential reduced risk of serious complications, and the possibility of an improved quality of life."
Ahmed Moussa, General Manager of General Medicines UK and Ireland for Sanofi, said: “One hundred years ago, the discovery of insulin revolutionised diabetes care.
“Today’s news marks a big step forward. We are now working with reimbursement bodies with the hope of securing timely access for eligible patients across England and Wales.”
Dr Elizabeth Robertson, Director of Research and Clinical at Diabetes UK, said: "Today's landmark licensing of teplizumab in the UK marks a turning point in the treatment of type 1 diabetes.
“For the first time, we have a medicine that targets the root cause of the condition, offering three precious extra years free from the relentless demands of managing type 1 diabetes.”
She added: “This is a breakthrough moment, but the next steps are critical. To ensure teplizumab reaches everyone who could benefit, we need it to be made available on the NHS, and the rollout of a screening programme to identify those with early-stage type 1 diabetes.
“Diabetes UK is leading the charge, driving type 1 diabetes screening initiatives, funding world-class immunotherapy research, and working with the NHS to make sure no one is left behind.
“Our vision is a future where multiple immunotherapies are available to stop type 1 diabetes in its tracks, at its earliest stages and beyond."
Today's announcement is a huge leap forward, but with teplizumab not yet available as standard on the NHS, there is much work to do before everyone in the UK who could benefit can access it.
Next, NICE must review teplizumab's cost-effectiveness to the NHS, and make recommendations about which patient groups should have access to it.
Crucially, for teplizumab to reach the people who could benefit from it, there also needs to be screening initiatives in place to identify people in the early, symptomless stages of type 1 diabetes.
Diabetes UK has been at the forefront of research into immunotherapies for type 1 diabetes for decades. This includes a major £2.8 million investment in 2015 to launch 30 clinical trial sites across the UK, accelerating the testing of immunotherapies at scale and speed.
At the same time, Diabetes UK has been leading efforts to make screening programmes for early-stage type 1 diabetes a reality. This includes funding vital research like the BOX study, ELSA study and UK Islet Autoantibody Registry. As well as working with the NHS to bring research advances into clinical practice.
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