NICE approves teplizumab, marking a new era in type 1 diabetes care

We are delighted with the decision by the National Institute for Health and Care Excellence (NICE) to approve teplizumab for use on the NHS. This is the first time a disease-modifying therapy for type 1 diabetes (T1D) will be available to eligible people across England and Wales.

Teplizumab is an immunotherapy that can delay the onset of symptomatic type 1 diabetes in people who are in stage 2 of the condition. This stage is identified through autoantibody screening and means the immune system has already begun attacking insulin-producing cells, but a person does not yet need insulin therapy or show symptoms.

A shift in the treatment of T1D

The availability of teplizumab represents a huge shift in how we understand and manage T1D, moving from reactive treatment at diagnosis to proactive intervention before symptoms begin.

Type 1 diabetes is an autoimmune condition that causes the body to attack and destroy insulin-producing cells in the pancreas. Without insulin converting food to energy, blood glucose levels rise dangerously, leading to serious complications and, without treatment, death. Diagnosis often comes suddenly and in crisis – one in four children in the UK are diagnosed in diabetic ketoacidosis (DKA), a life-threatening emergency caused by extremely high blood glucose levels.

A testament to science, persistence and community

Karen Addington MBE, Chief Executive of Breakthrough T1D, said: “This is a transformative moment for type 1 diabetes that gives people time, hope and options. For decades, a diagnosis of type 1 diabetes has meant immediate insulin therapy and lifelong management. Teplizumab changes that. It gives us a window to intervene earlier, reduce the risk of serious complications like diabetic ketoacidosis (DKA), and support people before symptoms begin.

“Breakthrough T1D has been at the forefront of this journey. We’ve funded the early research and advocated for access. Today’s decision is a testament to the power of science, persistence, and community.”

Parth Narendran, Professor of Diabetes Medicine at University of Birmingham, said: “Today’s NICE recommendation for teplizumab marks a significant milestone in the UK for people at the very earliest stages of type 1 diabetes.

“As the first disease-modifying therapy shown to delay progression to clinical, insulin-requiring type 1 diabetes, teplizumab has the potential to transform the treatment paradigm from reacting to disease onset to intervening earlier in the disease process. This means that patients identified early, for example through the ELSA study led by University of Birmingham, will benefit from treatment that can give valuable additional years free from the daily burden of managing type 1 diabetes. This decision will catalysing the development of screening, monitoring and prevention pathways that will underpin the future of type 1 diabetes care.”

How teplizumab works

Teplizumab is designed to target the immune system’s attack on insulin-producing beta cells in the pancreas. By modifying the immune response, it can delay the onset of clinical type 1 diabetes in people who are in stage 2.

Clinical trials showed that a single course of teplizumab delayed the onset of type 1 diabetes by an average of nearly three years, with some cases showing even longer delays.

Breakthrough T1D’s role in teplizumab’s journey

Here at Breakthrough T1D, we’ve played a pivotal role in the development of teplizumab, funding early-stage research, supporting clinical trials over the past 25 years and helping to secure approval for its use.

This investment helped prove the potential of immune intervention in T1D and paved the way for regulatory approval.

Teplizumab offers hope

Elena Boichak’s son Dima was found to be in the early stages of T1D through the ELSA screening study. Dima received teplizumab through the Early Access to Medicines Scheme (EAMS), which gives people with serious conditions access to medicines that have shown major benefits, before wider regulator authorisation has been granted.

Elena says: “We were contacted by Dr Renuka Diaz, who’s part of the early detection clinic in Birmingham. She told us about a new treatment, teplizumab, that was not yet available on the NHS but could be granted on compassionate grounds while the treatment was awaiting approval from NICE.

She asked whether we would like to consider it. I spoke at length with my sister, whose daughter was diagnosed when she was only seven. I also read and researched carefully. This wasn’t an easy decision. However, I believed in the science and if there was a chance we could delay the need for insulin, I thought it was worth it.

The treatment was delivered via infusion every day for two weeks. Each day began with checks before treatment could start. The infusion itself was relatively short, around 30 minutes, but we would have to stay for monitoring for a few hours after.

After treatment, we returned home and resumed normal life. Dima is now monitored locally every three months. So far, he remains symptom-free, and I’m hopeful this continues. If we hadn’t done it, Dima might have been diagnosed much later, possibly at a stage where he’d need urgent intervention, like my niece.”

Read more of Elena and Dima’s story

What NICE approval means

NICE’s decision means teplizumab will become available on the NHS in England and Wales for children over the age of eight and adults in stage 2 of type 1 diabetes.

We will continue to work with clinicians, researchers, and the T1D community to ensure equitable access across all four nations.

Next steps: national screening must follow

With teplizumab now approved for use on the NHS, our attention must turn to establishing national screening programmes to identify people at risk of, or in the early stages of type 1 diabetes.

Teplizumab is most effective before symptoms begin and insulin therapy is required. That means early detection is critical. We’re calling for coordinated, UK-wide screening to ensure eligible children and adults can benefit from this breakthrough therapy.

Without screening, most people will continue to be diagnosed too late to benefit from early intervention – often in emergency settings like diabetic ketoacidosis (DKA). We know that not everyone survives DKA. We now have a therapy that can change the course of type 1 diabetes. We must act to ensure it reaches the people who need it most.