The TGA’s approval of teplizumab marks Australia’s first step toward intercepting type 1 diabetes before it starts, but specialists warn that PBS listing and screening gaps must be addressed before the drug can reach those who need it most.

The Therapeutic Goods Administration approved the drug in late May for patients aged eight or older with stage 2 type 1 diabetes, delaying progression to a clinical diagnosis by an average of two years. It is the first new therapy approved for type 1 diabetes in a century.

The approval was welcomed by patient groups, clinicians and researchers. But access will hinge on a PBS listing and better screening, two leading endocrinologists told the limbic.

Professor Jane Holmes-Walker

“It is momentous for Australians that we can offer those at risk a treatment that can buy time without diabetes, or make management of T1D at diagnosis much less of a burden, potentially with once-daily injections and significantly less insulin in their first year,” said Professor Jane Holmes-Walker, senior staff specialist in diabetes and endocrinology at Westmead Hospital.

Teplizumab is not yet listed on the PBS. Manufacturer Sanofi said a funding submission would be considered at the July PBAC meeting.

“Cost is going to prevent all who would benefit from accessing the medication,” Professor Holmes-Walker said, noting the need for a PBS listing covering both newly diagnosed T1D and stage 2 disease, where the benefits were greatest.

Appropriate screening was the essential next step, she said. Most people who develop T1D have no family history, meaning a broad population screening programme was needed to identify those at risk early enough to benefit.

Professor Jennifer Couper, a paediatric endocrinologist at the University of Adelaide, said the approval came amid a wave of research into immune modulators for T1D.

“There are other trials going on now, to confirm that other immune modulators can delay or prevent type 1 diabetes,” she said.

She said immune therapy worked by dampening the immune attack on insulin-producing cells, preserving endogenous insulin production and making diabetes easier to manage.

Looking ahead, Professor Couper said combination approaches, pairing immune therapy with much lower doses of insulin, could reshape how T1D was managed.

Teplizumab targets a marker on T-cells, but Professor Couper said other immune cell types and even the insulin-producing cells themselves were targets under investigation. “There are lots of different targets, because the immune attack on the insulin-producing cell is complex,” she said.