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More evidence needed before new treatment to delay type 1 diabetes can be approved

The US Food and Drugs Administration (FDA) has decided more evidence is needed in order to approve a new, world-first treatment for type 1 diabetes. The immunotherapy drug – teplizumab – delayed type 1 diabetes by an average of three years in a clinical trial involving people who were at high risk of developing the condition. But researchers have been asked to collect more data on how the body processes the drug before it can be made available for the first time outside of clinical trials.

A breakthrough type 1 diabetes drug has faced a delay in becoming the world’s first new treatment since insulin to be licensed. The FDA decides if new, experimental treatments are safe and effective and should be given outside of clinical trials in the US.

They’ve been considering whether to approve the immunotherapy drug, teplizumab, for people who don’t yet have type 1 diabetes, but have a high risk of developing it in the future. 

After weighing up the evidence the FDA has decided that more data is needed to establish whether the newer commercial form of teplizumab is processed by the body in the same way as an earlier but very similar form of the drug which was used in previous clinical trials. Teplizumab has also been submitted for approval in the UK, but this review is at an earlier stage.

But it’s normal for regulatory bodies, like the FDA, to say they’d like to see more research and review again down the line once more evidence has become available. Licencing new treatments is a really rigorous process, which can take years. 

Teplizumab is the first immunotherapy for type 1 diabetes to go up for FDA review. It was granted ‘breakthrough therapy designation’ by the FDA, which happens when a treatment is considered to hold huge promise and means it is fast-tracked for review. 

Although it’s not been approved at this stage, the company who make teplizumab expects to be able to collect the extra information the FDA has asked for later this year. The decision to fast-track the review will also pave the way for other type 1 immunotherapy drugs – a number of which are being tested in clinical trials right now.

Chris Askew OBE, Chief Executive at Diabetes UK, said:

“It’s disappointing that teplizumab has not been approved in the US at this time, but we remain confident that with continued research, the world’s first licensed immunotherapy for type 1 diabetes is within reach.

“For 100 years, people living with type 1 diabetes have relied on insulin to treat the condition, and with immunotherapy, we’re now on the brink of being able to tackle the root cause of the condition – an immune system attack – for the first time. Immunotherapies for type 1 diabetes would be life-changing for people with or at risk of the condition, offering a way to slow or delay the condition for up to three years. 

“Preventing and curing type 1 diabetes is a key part of Diabetes UK’s mission. We’re funding immunotherapy research to help people at all stages of type 1 diabetes, and working with other organisations and key stakeholders to help make immunotherapies a reality in the UK. We hope that teplizumab and other immunotherapies will soon be approved, helping people living with or at risk of type 1 diabetes have a better chance of a healthier future.”

 

What is teplizumab?

Immunotherapies are new treatments that reprogramme the immune system so that it no longer attacks and destroys insulin-producing cells in the pancreas, dealing with the root cause of the condition for the first time.

Teplizumab is a type of immunotherapy drug called a monoclonal antibody. Monoclonal means that they are all copies of one type of antibody, and made in the laboratory. Antibodies recognise and bind to specific molecules in the body.

In the case of teplizumab, it recognises and exhausts killer T cells: a type of immune cell that is responsible for destroying insulin-producing beta cells in type 1 diabetes. This means that some beta cells are protected from the T cell attack, and people can keep on making enough of their own insulin for longer.

Scientists tested teplizumab in a landmark trial, funded by the National Institutes of Health and JDRF, involving 76 people aged 8-49 years who were at high risk of type 1. Those given teplizumab were less likely to be diagnosed with type 1 diabetes than a comparison group who were given a dummy (placebo) drug. Those that did develop type 1 diabetes during the study were on average diagnosed 3 years later than people who had received the placebo. 

This was the first study to show that it’s possible to intervene in the progression of type 1 diabetes in people at risk. And this matters – each day without type 1 diabetes counts.

A delay in diagnosis means people spend more years with their blood sugars in a healthy range and potentially could protect against long-term complications. It also means someone could get a diagnosis when they’re older and might be better equipped to manage their condition, perhaps avoiding childhood diabetes altogether.

Immunotherapy would be a huge shift in the way we treat type 1 diabetes – tackling the cause of the condition for the first time, instead of the symptoms. 

Finding people at risk of type 1 diabetes

Scientists have previously discovered that the immune attack behind type 1 diabetes can begin long before someone is diagnosed and that they can detect if someone is on the path to type 1.

In the years or decades before a diagnosis, the immune system starts to make proteins called autoantibodies. Scientists can use blood samples to look for autoantibodies to find out if someone is at risk of developing type 1 diabetes in the future.

Identifying who has autoantibodies before they have developed type 1 diabetes gives us a window of opportunity to use immunotherapy and disrupt the attack before it progresses too far. 

Autoantibody testing isn’t available in the UK at the moment. But close family members of people living with type 1 diabetes in the UK can sign up to a research project, called INNODIA, and if you’re eligible to take part you could be tested for autoantibodies.

What did the FDA say?

Provention Bio, the company who make teplizumab, submitted data to the FDA from studies when the drug was in an earlier but very similar form.

The FDA has asked for more evidence to show that the earlier drug and the newer commercial version of teplizumab are processed by the body in the same way.

They want to see more information that proves the similarity between the two before they can make a decision on whether to approve teplizumab. 

What’s next? 

While it’s disappointing that we’ll have to wait a little longer to see the world’s first ever immunotherapy licenced for type 1 diabetes, the FDA has left open the door for teplizumab and will review it again once more studies have been carried out. 

Provention Bio are running another clinical trial of teplizumab right now involving people recently diagnosed with type 1 diabetes, to see if the treatment could also help at this stage.

They hope that this study will provide the extra data they need later this year. Once the researchers have analysed these results, it is likely they will submit them to the FDA, who will consider whether teplizumab should then be licenced. 

The FDA only decides what should happen in the US. For a new drug to be licensed in the UK, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) would need to approve it as safe and effective.

Provention Bio has submitted an application to the MHRA for teplizumab. The MHRA has awarded teplizumab an ‘Innovation Passport’ under the ‘Innovative Licensing and Access Pathway’. This pathway is designed to help speed up access to promising new medicines in the UK. Teplizumab is one of the first drugs to be given this special status. It means the MHRA will accelerate its assessment of teplizumab and that other important organisations, like the NHS and the National Institute for Clinical Excellence (NICE), will be involved in the process from the start. MHRA’s assessment is at an early stage and we’ll update you when we learn more about possible timelines.   

In the meantime, we’re working hard to make sure immunotherapies become a reality in the UK by funding more research, including the UK’s type 1 diabetes immunotherapy consortium in partnership with JDRF, and working with key stakeholders to help make sure these treatments become available in the NHS, once approval is secured. 

Could immunotherapy help people already living with type 1 diabetes?

The FDA was deciding whether to approve teplizumab for people who have a higher risk of getting type 1 diabetes.

But teplizumab and other immunotherapies are also being tested right now in people who are newly diagnosed with type 1 diabetes to see if they can help to protect their remaining beta cells. The more we can protect, the more insulin you can produce on your own. 

Find out more about how immunotherapies work and who they could help

In the future, immunotherapies might form a cure for people who have been living with type 1 diabetes for a longer time. Our scientists are looking for ways to replace or regenerate the beta cells that have already been destroyed.

Once we can do this, immunotherapies could potentially be used alongside the beta cell replacement therapy to protect new cells from another immune system attack.

How you can get involved

Breakthroughs can only happen thanks to the people who take part in research and you could play an important role in advancing immunotherapy research. 

  • If you’re newly diagnosed with type 1 diabetes, you could be eligible to take part in an immunotherapy trial within 100 days of your diagnosis. Sign up to the type 1 diabetes immunotherapy consortium or ADDRESS-2 to find out about opportunities. 
  • If you’re a close relative of someone with type 1 diabetes, you can sign up to INNODIA
  • If you already have type 1 diabetes, look for research opportunities on our Take Part in Research page.

Research also can’t happen without funding. It’s only with your help that our scientists can push ahead. By making a donationadopting one of our research projects or fundraising for us, you can help us reach the next breakthrough.

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