Teplizumab: The world’s first type 1 diabetes disease-modifying drug approved in the US

What is teplizumab?

Teplizumab is an immunotherapy drug proven to delay the onset of type 1 by up to three years in high-risk individuals. It effectively tackles the root cause of the condition for the first time. The drug also has the potential to slow progression of type 1 in the long-term.

Professor Kevan Herold, a JDRF-funded researcher who has been developing teplizumab for nearly 30 years, said: “This decision represents a turning point in the field. First, it identifies a way in which an immune therapy to stop the disease process might be combined with cell replacements in those with type 1 diabetes. It also suggests that it is time to more broadly screen to identify those at risk of type 1 since now there is a therapy that can change its course.”

Why is this delay important?

For children at risk of type 1 who are approaching adolescence, delaying type 1 would enable the pancreas to grow to adult size, giving better disease outcomes.

Teplizumab can also help prevent traumatic and potentially fatal diabetic ketoacidosis (DKA) in individuals at risk of type 1. DKA is a severe lack of insulin which leaves the body unable to use glucose for energy. As a result, it starts to use fat instead, releasing chemicals called ketones which turn the blood acidic. One in four children in the UK experience DKA when diagnosed.

Beth Baldwin – whose son Peter died of DKA, aged 13 – said: “The potential for a ground breaking new drug to delay the onset of type 1 diabetes will be life changing for so many. The ripple effect this will have will improve diagnosis outcomes and even buy precious time whilst the search for a cure is ongoing – a hopeful reality in our lifetime. My son Peter never had this opportunity. To have teplizumab licensed in the UK will be a giant step forward.”

Teplizumab would not exist without JDRF

We have been instrumental in the development of teplizumab by funding the initial exploratory research, trial recruitment networks, and the clinical trials themselves.

Professor Kevan Herold said: “The story with the clinical use of teplizumab began with a JDRF grant to support a trial in patients with new onset type 1 diabetes more than two decades ago. The success of this initial study planted a seed that led to further studies and support from the National Institute of Health. Over the years the payback on this initial investment has been enormous. Not only for bringing the drug to the current consideration for approval but also teaching us about the immune and beta cell dysfunction that causes the disease and suggesting multiple strategies for preventing, delaying, and even eliminating type 1 diabetes.”

Karen Addington, CEO of JDRF UK, said: “This landmark approval is tremendously important and we, JDRF, are proud to have funded the research into teplizumab from its start nearly thirty years ago. The world now has a drug which is proven to effectively tackle the root causes of type 1 diabetes, delaying the onset of the condition and slowing down disease progression. Our work now focuses on securing approval in the UK. We will also be leading further research into the future potential of teplizumab to entirely prevent type 1, helping to eradicate this condition from everyone’s lives.”

How does teplizumab work?

The drug helps to protect insulin-producing beta cells in the pancreas, by attaching markers to the cells in the immune system that go awry, and attack and destroy beta cells in type 1 diabetes. These markers neutralise the rogue cells, enabling beta cells to live and produce insulin. Research is showing that teplizumab can also slow disease progression among newly diagnosed people with type 1 diabetes.

Teplizumab in the UK

The FDA’s approval of teplizumab will pave the way for approval in the UK. Here, the drug has already been awarded a favourable ‘innovation passport’ status by the UK medicines and healthcare regulator, the MHRA.

Screening for type 1 is vital

Professor Colin Dayan, who leads the UK Type 1 Diabetes Immunotherapy Consortium, said: “This is very exciting news and a key first step in transforming how we care for type 1 diabetes. The introduction of this treatment adds weight to the value of screening for type 1 diabetes. This will lead to many more people being identified who are in the earliest stages of losing their insulin-producing beta cells. This could prevent up to a thousand people a year from having emergency hospital admissions to start insulin. Identifying these people will also allow much more rapid testing of other treatments in addition to teplizumab so that we can delay the need for insulin for many more years. Ultimately, I hope it will lead to needing insulin to treat type 1 diabetes in childhood being a thing of the past.”