The news that the FDA has decided not to license the immunotherapy teplizumab at this time is disappointing for those of us who have been following this drug’s story closely.
One hundred years since the discovery of insulin, we are still waiting for a treatment that can actually change the course of type 1 diabetes, not just treat the symptoms.
Today, type 1 diabetes remains one of the few autoimmune conditions that does not have an approved immunotherapy.
But we needn’t be downhearted – there are plenty of reasons to remain hopeful.
Firstly, although this news wasn’t exactly what we hoped for, it is not the end for teplizumab.
According to the drug’s manufacturer, Provention Bio, the FDA did not raise any concerns around the drug’s safety or efficacy in delaying the progression of type 1 diabetes.
Instead, it asked for more evidence that the drug used in clinical trials worked in a comparable way to the product being licensed.
This was expected, and Provention Bio has already stated that it will work to collect this data through the PROTECT trial, which is running in the UK – thanks to the JDRF co-funded Type 1 Diabetes UK Immunotherapy Consortium.
Teplizumab blazing a trail
What’s more, even though teplizumab is the first immunotherapy for type 1 diabetes to be reviewed by the FDA, it is just one of many such potential therapies we’re advancing through research funding and advocacy.
Several of these immunotherapies are being trialled here in the UK, again thanks to the Type 1 Diabetes Immunotherapy Consortium.
Crucially for this research, teplizumab showed that it is possible to delay the onset of type 1 diabetes. It proved that the right drug can target and disrupt the immune attack at the heart of type 1.
This is vital for any therapy aimed at curing or preventing type 1.
Lastly, teplizumab has also blazed a trail for these immunotherapies still to come.
It showed that the FDA is willing to consider licensing therapies that change the course of type 1 diabetes before it is even diagnosed. This is commonplace for many other conditions – doctors routinely prescribe statins and blood pressure medication to ward off serious heart problems – so why not type 1?
By considering preventative immunotherapies, the FDA is opening the door to treatments that give people extra years without fingerprick checks, carb counting and insulin therapy.
We now expect the FDA and the drug’s manufacturer, Provention Bio, will continue working together to bring teplizumab to people with, or at risk of developing, type 1 diabetes in future.