The Food and Drug Administration has
FDA officials said the injectable drug, teplizumab, can postpone the onset of type 1 diabetes by at least 2 years.
The drug, known by the brand name Tzield, is available for adults and children 8 years and older who currently have stage 2 type 1 diabetes.
It is administered by intravenous infusion once a day for 14 consecutive days.
It’s not recommended for people with insulin-dependent stage 3 type 1 diabetes or for people diagnosed with type 2 diabetes.
“Today’s approval of a first-in-class therapy adds an important new treatment option for certain at-risk patients,” said Dr. John Sharretts, the director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA’s Center for Drug Evaluation and Research, in a statement. “The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease.”
FDA officials said Tzield works by binding to certain immune cells.
“Tzield may deactivate the immune cells that attack insulin-producing cells while increasing the proportion of cells that help moderate the immune response,” the FDA officials explained.
Members of the diabetes community said the new drug could be a game changer.
“This approval is a watershed moment for the treatment and prevention of type 1 diabetes,” Dr. Mark Anderson, director of the University of California San Francisco Diabetes Center, told ABC News. “Until now, the only real therapy for patients has been a lifetime of insulin replacement. This new therapy targets and helps to halt the autoimmune process that leads to the loss of insulin.”
In May 2021, an FDA advisory committee recommended teplizumab be approved by the full agency.
In June 2022, the FDA opted to delay approval of the drug while its manufacturer, Provention Bio, retooled the medication.
“I will say with confidence that years from now, teplizumab will widely be seen as revolutionary, and in fact for some, including myself, I have already seen it in such a light,” Dr. Mark Atkinson, American Diabetes Association Eminent Scholar for Diabetes Research and director of the University of Florida Diabetes Institute, wrote in his testimony to the FDA earlier this year.
He called teplizumab the most impactful diabetes breakthrough since home blood glucose meters replaced urine testing.
“We know this can have a blockbuster effect on the pre-diagnosed,” said Frank Martin, PhD, the director of research at JDRF, a global research and advocacy organization for type 1 diabetes.
Teplizumab is an anti-CD3 monoclonal antibody drug that binds to the surface of T-cells in the body and helps suppress the immune system.
Similar drugs are being tested for the treatment of other conditions such as Crohn’s disease and ulcerative colitis.
After a median follow-up of 51 months, researchers reported that 45% of the 44 people who were given Tzield were later diagnosed with stage 3 type 1 diabetes. That compared with 72% of the 32 people who received a placebo.
Researchers noted that the mid-range time between administration of the drug and stage 3 diagnosis was 50 months for people who received Tzaid and 25 months for those who were given a placebo.
The most common side effects were decreased levels of certain white blood cells, rashes, and headaches.
Teplizumab was born from a long line of drugs created and tested over more than three decades.
The idea took root in the labs of Dr. Kevan Herold and Dr. Jeffrey Bluestone at the University of California San Francisco.
It was in 1989, when working with cancer patients, that Bluestone realized an anti-CD3 drug could be a key in stopping the progression of type 1 diabetes because of how it helped transplant patients.
His theory seemed to hold up in small studies.
Since type 1 diabetes manifests when a person’s immune system gets confused and attacks their insulin-producing beta cells rather than protecting them, Bluestone surmised that by creating monoclonal antibodies in a lab that can be introduced into the body of a person on the verge of developing type 1 diabetes, those will bind to the CD3 cells that are attacking the beta cells and stop the attack.
Over the years, researchers like Herold and Bluestone, along with companies like Tolerx, worked to find just the right level of anti-CD3 to make that effort a success.
Tolerx came close to approval of its drug about 10 years ago, but it did not make it past phase 3 trials with the FDA due to some significant side effects of flu-like symptoms.
Other trials fell short as well, which often happens as drug research progresses.
Four years ago, Provention Bio picked up the research and pushed it along. They were frustrated with how the medical system handles diagnoses of autoimmune diseases in general, said Ashleigh Palmer, the co-founder and chief executive officer of Provention.
“The medical system waits for patients to exhibit symptoms. Very often, by that point, irreversible damage has been done,” Palmer told DiabetesMine.
“Can you imagine,” he added, “a system in which a patient with kidney disease presents at the point of dialysis? Insulin therapy is pretty much the same as if we did that. We go right to the [intense and chronic] treatments at the start.”
At the point where Provention Bio took over, the global type 1 diabetes screening research collaboration TrialNet was pumping some decent study participant numbers into the project. More than 800 patients have received the treatment in multiple studies to date.
With the work done over those past decades, it seemed they had found what Palmer calls the “Goldilocks” formula for the medication.
“Not too little immune response alteration and not too much; just the right amount,” he said.
Katie Killilea of Rhode Island told DiabetesMine that her son entered the Teplizumab trial at Yale in 2013 after she and her son were both tested via TrialNet at her other son’s diabetes camp.
Killilea herself was diagnosed shortly after. But her son, who was farther back in the progress toward type 1 diabetes, was able to remain in the study.
The challenges, she said, were that her son [along with his dad] had to spend 3 weeks up near Yale, a bit of a bump in the life of a 12-year-old, and a difficult setup for most families.
“It gives me hope, but the whole time [in 2013], I felt acutely aware of how difficult the teplizumab trial was for families financially,” she said.
“You had to have a parent who could take time off from work, another parent to stay home with the other child or children. It seemed unrealistic for us, and maybe impossible for others to participate,” she said, stressing that these issues need to be worked out.
But the benefits were many, she said.
“Since he had the drug, his blood glucose returned to normal for a while. TrialNet did glucose tolerance tests every 6 months,” she said.
And when the time did come that her son developed type 1 diabetes, Killilea found it to be a more manageable transition as opposed to her other son’s previous diagnosis.
“Although he was not able to keep type 1 diabetes at bay forever, he did have a very gentle landing and was diagnosed with type 1 diabetes before he needed to use insulin,” she said.
Our experts continually monitor the health and wellness space, and we update our articles when new information becomes available.
Nov 18, 2022
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Jul 3, 2021
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