Britian recommends teplizumab to delay type 1 diabetes
Britain has become the first country in Europe to recommend teplizumab to delay progression of type 1 diabetes.
Results from the TN-10 phase 2 trial involving patients aged 8 and older showed a median delay of 24.6 months in the primary analysis and up to 32.5 months with extended follow-up compared to placebo.
The treatment is administered over a 14-day intravenous course and has a manageable safety profile, with most adverse effects being transient or mild.
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A therapy that can delay the onset of type 1 diabetes (T1D) by an average of nearly 3 years has been recommended for NHS use in England — the first country in Europe to do so through a health technology appraisal.
The National Institute for Health and Care Excellence (NICE) has recommended teplizumab (Tzield, Sanofi) for patients aged 8 years or older with stage 2 (presymptomatic) disease, in what Diabetes UK describes as “a new age of type 1 diabetes treatment.”
”For the first time in 100 years, we are moving beyond insulin, with a medicine that targets the root cause of the condition,” said Dr Elizabeth Robertson, director of research and clinical at Diabetes UK, in a press release. ”This is an extraordinary moment for celebration in the type 1 diabetes community.”
The recommendation, issued as final draft guidance, is conditional on the manufacturer offering the medicine at a commercially agreed discount.
Key Points
NICE recommends teplizumab for stage 2 T1D, age ≥8 years.
First European NHS appraisal for delaying presymptomatic T1D progression.
TN-10: median stage 3 delay 24.6 months; extended follow-up 32.5 months.
Once-daily IV x14 days; rash 36%, transient lymphopenia, infections similar.
Implementation needs autoantibody screening, secondary care infusion, follow-up; ~1100 eligible year 1.
Which biomarkers predict teplizumab response in stage 2 T1D?
How does teplizumab compare with other T1D prevention strategies?
What screening pathways best identify presymptomatic T1D early?
Addressing an Unmet Need
T1D progresses through three stages: Stages 1 and 2 are asymptomatic but are defined by the presence of two or more pancreatic islet autoantibodies, while stage 3 marks the onset of symptomatic disease requiring lifelong insulin therapy. Currently, most patients receive a diagnosis only at stage 3, when significant beta-cell loss has already occurred.
Teplizumab is an Fc receptor-nonbinding anti-CD3 monoclonal antibody designed to modify T lymphocytes involved in destroying insulin-producing beta cells.
Lucy Common, clinical nursing advisor at NICE, said: “Having time before the onset of stage 3 diabetes is not just a clinical benefit; it can make a meaningful difference to people’s lives, their mental well-being, and the wellbeing of the families and carers who support them. It is encouraging to see a treatment that has the potential to be made available across the NHS.”
Delays Progression to Stage 3 Disease
The recommendation is based on findings from TN-10, a phase 2 trial involving 76 participants aged 8 years or older with stage 2 T1D. In the primary analysis, with a median follow-up of 24.5 months, median time to stage 3 onset was 49.5 months with teplizumab vs 24.9 months with placebo, a delay of 24.6 months.
In extended follow-up, median time to stage 3 onset was 59.6 months in the teplizumab group compared with 27.1 months in the placebo group, a difference of 32.5 months.
Helen Knight, director of medicines evaluation at NICE, said it was a “genuinely exciting recommendation” based on “the best available evidence, striking a balance between clinical benefit and value for the taxpayer.”
Dosing, Administration, and Safety
Teplizumab is administered as a once-daily 14-day intravenous course in hospital. The dose is escalated from 51 μg/m2 on day 0 to 103 μg/m2 on day 1, 207 μg/m2 on day 2, and 413 μg/m2 on day 3, before reaching the maintenance dose of 826 μg/m2 on days 4 through 13.
The safety profile was broadly manageable. A spontaneously resolving rash occurred in 36% of teplizumab recipients. Transient lymphopenia was observed, with lymphocyte counts reaching a nadir on day 5 and recovering by day 45 in all but one participant. Infection rates were comparable between treatment groups.
Cost and Implementation
Teplizumab has a list price of £10,939.12 per 2-mg vial, excluding VAT, although the NHS will receive the drug at a discounted price under the commercial agreement. Deploying teplizumab in practice will require significant infrastructure development.
There is currently no national pathway for identifying presymptomatic T1D, and delivering this treatment will necessitate standardised autoantibody screening, secondary care administration facilities, dedicated follow-up protocols, and staff training. In the interim, patients are likely to be identified through research studies such as the ELSA study, which screens children aged 2-17 years for early-stage T1D, and the T1DRA study, which screens adults aged 18-70 years. Clinical experts estimate that each tertiary centre will treat approximately four to five patients annually.
NICE estimates that approximately 1100 people could be eligible for treatment with teplizumab in the first year, of whom around 555 are expected to take up the offer. This figure is higher than in subsequent years because it includes people already identified through current screening trials who are waiting for a treatment option. From year three, the eligible population is expected to stabilise at around 820 people annually, with approximately 490 expected to take up treatment.
From www.medscape.com
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