The US Food and Drug Administration (FDA) has decided not to license the immunotherapy teplizumab at this time, but has left the door open for its approval in future.
The manufacturer of teplizumab, Provention Bio, had asked the FDA to license the drug for delaying type 1 diabetes in people at risk of developing the condition.
This followed clinical trials where teplizumab was shown to delay the onset of type 1 diabetes – and a life of injecting insulin – for an average of three years.
However, the FDA said that Provention Bio needed to provide more evidence that the drug used in these clinical trials worked in a comparable way to the product they wanted a licence for.
This decision was expected, as the FDA had previously asked for more evidence of this comparability. Crucially, Provention Bio reported that the FDA did not mention any concerns around the drug’s safety or efficacy in delaying the progression of type 1 diabetes.
Provention Bio has already stated that it will work to collect more comparability data through the PROTECT trial, which is running in the UK thanks to the JDRF co-funded Type 1 Diabetes UK Immunotherapy Consortium.
In a statement, Provention Bio pledged to “work collaboratively with the FDA to hopefully secure approval of teplizumab and bring the first disease-modifying therapy for [type 1 diabetes] to at-risk patients as soon as possible.”
If teplizumab is subsequently licensed, it would mean that, for the first time, we could delay the onset of type 1 diabetes for those at risk – and it could pave the way for future life-changing treatments for those already living with the condition.
Door still open to teplizumab
Although teplizumab is the first drug to be reviewed by the FDA for its potential to change the course of type 1 diabetes, it is just one of many such potential therapies JDRF is advancing through research funding and advocacy.
Several of these immunotherapies are being trialled in the UK, also through the Type 1 Diabetes Immunotherapy Consortium.
Conor McKeever, Research Communications Manager at JDRF, said: “Although the FDA has not licensed teplizumab at this time, the door to approving this therapy for delaying type 1 diabetes is still very much open.
“This ongoing consideration by regulators demonstrates exciting progress in type 1 diabetes research and we look forward to more news on teplizumab and other immunotherapies soon.
“Therapies that can change the course of type 1 diabetes are vital to addressing the unmet needs of people with, or at risk of the condition. We expect the FDA and Provention Bio will continue working together to bring teplizumab to people at risk of developing type 1 .
“In the meantime, we will continue to research new therapies that put us on the path to preventing and ultimately curing type 1 diabetes.”