U.S. regulators have approved Tzield (teplizumab-mzwv) as the first-ever treatment to delay the onset of clinical Type 1 diabetes (T1D).
The decision by the U.S. Food and Drug Administration was hailed by diabetes experts as “a historic moment” and “the start of a seismic shift” in how T1D is treated.
The first-in-class immunotherapy from U.S. biopharma Provention Bio Inc. has been heralded as the most important breakthrough for the condition since insulin was discovered 100 years ago.
Intravenous infusions once daily for 14 days will now be marketed in the U.S to delay the onset of stage 3 T1D in adults and children aged 8 years and older who currently have the stage 2 condition.
This follows a trial in which Tzield delayed the median onset of stage 3 T1D by approximately two years compared with placebo in those with stage 2 T1D.
Pricing for the drug is expected to be announced imminently. Provention Bio last month announced a co-promotion agreement with pharma giant Sanofi for its U.S. launch.
The landmark approval means that, for the first time, the immune system attack that is the root cause of the T1D can be addressed.
Tzield binds to and deactivates immune cells that attack insulin-producing cells and increases the proportion of cells that help moderate immune response.
John Sharretts, director of the diabetes, lipid disorders, and obesity division in the FDA’s Center for Drug Evaluation and Research called it “an important new treatment option” for certain at-risk patients.
He added: “The drug’s potential to delay clinical diagnosis of [T1D] may provide patients with months to years without the burdens of disease.”
An estimated 1.6 million people in the U.S already live with T1D, with more than 64,000 new cases diagnosed each year.
The incidence is increasing, with numbers expected to top five million within the next few decades. It is one of the most common chronic diseases to develop in childhood.
“People with T1D require life-long insulin replacement therapy, so to be able to provide an individual with a two-year delay from the symptoms and burden of T1D is a tremendous accomplishment as we look towards finding a cure,” said American Diabetes Association chief scientific and medical officer Robert Gabbay.
The anti-CD3 monoclonal antibody Tzield was evaluated in a randomized trial of 76 patients with stage 2 T1D. Participants randomly received intravenous infusion with the drug or placebo for 14 days.
Over a median of 51 months’ follow up, 45% of the 44 patients who received Tzield were later diagnosed with stage 3 T1D versus 72% of the 32 placebo-treated patients.
The mid-range time from randomization to the diagnosis of stage 3 T1D was twice as long for patients receiving Tzield compared with placebo, at a corresponding 50 versus 25 months, a statistically significant difference.
The most common side effects included lymphopenia, rash, leukopenia and headaches.
Provention Bio aims to tackle chronic autoimmune conditions by seeking out asymptomatic disease and genetic risk early on and attempting to intercept it.
It is also developing an investigational polyvalent coxsackievirus B vaccine for the prevention of T1D and other candidate drugs for lupus and celiac disease.
