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FAQs about teplizumab

Our answers to frequently asked questions about teplizumab, the first disease-modifying drug for type 1 diabetes.
Content last reviewed and updated: 27.03.2024

What is teplizumab?

Teplizumab is an immunotherapy drug, which has just been approved by the USA’s Food and Drug Administration (FDA) to delay type 1 diabetes. It helps to protect the insulin-producing beta cells from the immune attack that causes type 1 by attaching to markers on a type of immune cell called T-cells. Teplizumab has been shown to delay the development of type 1 in at-risk individuals by an average of three years.

Why is teplizumab important?

Teplizumab is the first disease-modifying therapy available to delay clinical type 1 in individuals at high risk anywhere in the world. The approval of teplizumab proves to funders of research in government and industry that it is possible to develop drugs for type 1. This should lead to an increase in funding for type 1 diabetes research and hopefully bring about more treatments.

Why is a delay in type 1 diabetes diagnosis important?

A delay in clinical type 1 diabetes of at least two years is clinically meaningful. Any time period that a person does not have to be dependent on insulin means they are avoiding important risks such as hypoglycemia and the significant constant burdens of life with type 1. This can also play a key role in preventing complications such as eye, kidney, and heart diseases.

How does the FDA approval affect people in the UK?

The FDA is the largest global medical licensing agency, so often drug manufacturers apply to the FDA first to approve their new drug. If the FDA approves a drug, it is likely that the MHRA (who approve drugs in the UK) and the EMA (who approve drugs in Europe) will do so too. Both the MHRA and the EMA have granted teplizumab procedures to fast-track them through the approval process.

If MHRA approve teplizumab, when can I get it?

The approval agencies just address the safety of the drug. For teplizumab to be adopted in the UK, NICE must deem it clinically cost effective and adopt it under the NHS. There is lots of positive evidence for this, but the logistics of clinical delivery must also be worked out. An education programme for doctors needs to be developed to teach them how to manage people who aren’t yet diagnosed with type 1. Dr Rachel Besser is working on this – read her blog post.

How can I find out if I’m at risk of developing type 1?

There are trial screening programmes in the UK that test children’s blood for markers of the very early stages of type 1. One of these is called the ELSA study and screens children aged 3-13 years across the UK. Every child between that age bracket is eligible to be screened.

How does this affect people who already have type 1?

Researchers are also testing teplizumab in people recently diagnosed with type 1 via the PROTECT study. The study was possible thanks to the JDRF co-funded UK T1D Research Consortium, which recruits people with type 1 into UK clinical trials of new immunotherapy treatments. Recruitment for this study has now closed, but you can use our clinical trials finder to see which other studies you are eligible for.

Were there any negative side effects in the clinical trials?

In the clinical trial under review, some people who took teplizumab developed a rash and had a temporary lower level of white blood cells than they should have. White blood cells are cells within the immune system that help fight off infectious diseases. Crucially, the participants’ white blood cell count soon returned to normal.

Are other similar treatments being developed?

Other drugs that target the immune system – known as immunotherapy drugs – are being developed and tested on people with type 1. An example of this is ustekinumab, a drug licensed to treat other autoimmune conditions including psoriasis, which is being trialed in children recently diagnosed with type 1. The approval of teplizumab sets a precedent that immunotherapies are a viable treatment for type 1. This encourages pharmaceutical companies to invest in them and paves the way for more drugs to become available.

How did JDRF support the development of this drug?

As part of our mission to accelerate life-changing breakthroughs, JDRF played a key role in funding early-stage research and facilitating regulatory pathways to help bring teplizumab to market. As well as co-funding the UK T1D Research Consortium, JDRF has funded international clinical networks that supported early clinical trials to determine who would benefit from teplizumab and whether the drug works for people both with and at high risk of type 1.

We have continuously funded a researcher called Professor Kevan Herold, who initially showed that an immunotherapy drug could prevent type 1 in mice. This drug later became a version that could be used in humans: teplizumab. Professor Herold was the lead on the first ever study to effectively delay the onset of type 1, which formed the basis of the FDA approval application. He is still receiving JDRF funding to explore the biological mechanisms through which teplizumab delays type 1 development.

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