FDA approves first drug that can delay onset of type 1 diabetes

A “life-changing” immunotherapy drug proven to delay the development of type 1 diabetes has been given the green light by an official regulatory board in the United States. 

The U.S. Food and Drug Administration (FDA) has approved teplizumab to delay the onset of type 1 diabetes in adults and children aged eight years and older.

Otherwise known as Tzield, the FDA-approved drug binds to certain immune system cells and delays progression to stage 3 type 1 diabetes.

According to the regulators, teplizumab may deactivate the immune cells that attack insulin-producing cells, while increasing proportion of cells that help moderate the immune response. Teplizumab is administered by intravenous infusion once daily for 14 consecutive days.

Dr John Sharretts, director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA’s Center for Drug Evaluation and Research, said: “Today’s approval of a first-in-class therapy adds an important new treatment option for certain at-risk people.

“The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide people with months to years without the burdens of disease.”

Teplizumab’s safety and efficacy were evaluated in a randomized, double-blind, event-driven, placebo-controlled trial with 76 people with stage 2 type 1 diabetes.

In the trial, participants randomly received teplizumab or a placebo once daily via intravenous infusion for 14 days.

The primary measure of efficacy was the time from randomization to development of stage 3 type 1 diabetes diagnosis.

The trial results showed that over a median follow-up of 51 months, 45 per cent of the 44 people who receivedteplizumab were later diagnosed with stage 3 type 1 diabetes, compared to 72 per cent of the 32 patients who received a placebo.

The mid-range time from randomization to stage 3 type 1 diabetes diagnosis was 50 months for those who received teplizumab and 25 months for those who received a placebo. This represents a statistically significant delay in the development of stage 3 type 1 diabetes.

Beth Baldwin, whose son 13-year-old son Peter died after a diabetic ketoacidosis emergency in 2014, said: “A drug like this would be life-changing.

“You cannot stop people getting type 1 diabetes for now, but delaying the onset would be phenomenal, particularly for children.”

She added: “It means three years of not having to intensively manage the condition, and it may delay it long enough for more research to take place. It is a huge step forward.”

Chris Askew OBE, Chief Executive at Diabetes UK said: “This landmark approval of teplizumab in the US is the start of a seismic shift in how type 1 diabetes is treated. For 100 years, people living with type 1 diabetes have relied on insulin to treat the condition, and this decision means that for the first time, the root cause of the condition – an immune system attack – can be tackled, and type 1 diabetes potentially delayed for up to three years.

“The licensing of teplizumab in the UK must now be accelerated, and we’re working with the NHS and with other diabetes charities and key stakeholders to ensure that people in the UK can benefit from this life-changing treatment as soon as possible. Preventative treatments for type 1 diabetes can only be effective when combined with screening programmes to identify those at risk, and the launch of the ELSA screening trial this week is perfectly timed to help unleash the benefits of teplizumab here in the UK.”

He added: “Diabetes UK is funding immunotherapy research to help people at all stages of type 1 diabetes, and we hope this monumental breakthrough will open the door for increased research investment, to develop further effective immunotherapies to treat the condition.

“This is a significant moment in the history of type 1 diabetes, and one that will shape the future – propelling us closer to the day where type 1 diabetes can be prevented or cured altogether.”

 Rachel Connor, from the JDRF UK charity, said: “This is a game-changer. To me this is the start of a new era for the treatment of type 1 diabetes.

“It is the first time we are able to get to the heart of why the condition develops and help change the process, so we are not just treating the symptoms anymore. Once we can do that, we can find other ways to do it better and for longer.”