FDA approves drug to delay type 1 diabetes: A watershed moment for the T1D community – The Business Journals

On Nov. 17, the U.S. Food and Drug Administration (FDA) weighed the evidence for teplizumab to delay type 1 diabetes (T1D) in at-risk individuals, and the T1D community got a big win: approved, under the brand name Tzield™.
Type 1 diabetes is an autoimmune disease in which a person’s pancreas stops producing insulin, a hormone that enables people to get energy from food. It occurs when the body’s immune system attacks and destroys the insulin-producing cells in the pancreas, called beta cells. While its causes are not yet entirely understood, scientists believe that both genetic factors and environmental triggers are involved. Its onset has nothing to do with diet or lifestyle. There is nothing you can do to prevent T1D, and — at present — nothing you can do to get rid of it.
Tzield (teplizumab-mzwv) is the first disease-modifying therapy for individuals at-risk for developing T1D. (At-risk means they exhibited two or more T1D-related autoantibodies — antibodies against one’s own self — and their blood glucose is starting to be abnormal, but they have not yet become insulin dependent.)
For the first time in history, there is an approved therapy that addresses the autoimmunity behind T1D, not just the symptoms that it causes in 1.4 million Americans.
Aaron Kowalski, Ph.D., CEO of JDRF, celebrated the decision — the first approved therapy that can slow, halt or reverse the course of the disease — for T1D.
“A delay in the onset of type 1 diabetes will have a tremendous impact on the daily lives of people at risk for diabetes, their families and the overall health system,” said Kowalski. “It would free them from the constant burden and stress of blood sugar monitoring and insulin administration. It would free them from the worry and fear of short- and long-term complications, while giving them the opportunity to learn more about disease management. That is clinically meaningful.”
Tzield can start to address the stark unmet need for disease-modifying therapies and help people at-risk for T1D and their families avoid the burden and complications this disease brings. Tzield, and other potential disease-modifying therapies that JDRF has invested research into, put us on the critical pathway to finding cures with the hope of preventing T1D entirely.
JDRF thanks the FDA for their thorough and thoughtful review of the evidence and data, showing that the benefits outweigh the risks to delay clinical T1D in at-risk individuals.
What it means for the community
A delay in onset will be a game changer for at-risk people. Dr. Cory Wirt has experienced this firsthand. She enrolled her daughter Claire, who had biomarkers and was at-risk for developing T1D, in a clinical trial for Tzield seven years ago. Today, she has yet to progress into clinical T1D.
“As a mom, I appreciate 83 months of not checking blood-sugars multiple times per day, worrying about life threatening lows and balancing my child/teen’s independence with the importance of tight medical control,” said Wirt. “Not to mention the significant cost of supplies, office visits and emotional stress. We don’t know how long the effects of the treatment will last, but every day without insulin has been a gift.”
Tracy Olsten is another parent who made the decision to enroll her daughter Mikayla in clinical trials for Tzield. “We know Mikayla has the biomarkers,” said Olsten. “We know the probability is high that she will develop T1D in her life. But (Tzield gives her) a little bit more time to live her teenage, young adult and adult years without injections and finger pokes.”
A day decades in the making
Today’s decision would simply not have been possible without decades of JDRF work, from funding discovery research to clinical development to work with regulators. JDRF had a hand in the development of Tzield from the very beginning.
The JDRF T1D Fund made a strategic investment in Provention Bio in 2017 that brought the company into T1D for the first time. That investment has helped catalyze hundreds of millions of dollars toward the clinical development, regulatory work and launch preparation that made this moment possible.
Herold is one of the pioneers who began this research. Here are his thoughts on this momentous day:
“The story with the clinical use of teplizumab began with a JDRF grant to support a trial in patients with new onset type 1 diabetes more than two decades ago,” said Herold, a professor at Yale School of Medicine. “The success of this initial study planted a seed that led to further studies and support from the NIH.
“The recent decision represents a turning point in the field. First, it identifies a way in which an immune therapy to stop the disease process might be combined with cell replacements in those with type 1 diabetes. It also suggests that it is time to more broadly screen to identify those at risk for type 1 diabetes, since now there is a therapy that can change its course.”
The success of Tzield — the first disease-modifying drug for T1D — is something the T1D community, JDRF and other like-minded nonprofits, companies, consortia and regulators should be gratified and proud of. But our work to find cures continues full steam ahead. We have multiple cure therapies in the pipeline today whose path to market became a little clearer with this approval. We are moving ever closer to a world without this disease, and Tzield receiving approval is one gigantic step along the way.
Donate to JDRF today.
JDRF is the leading global organization funding type 1 diabetes (T1D) research. Our mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. Our staff and volunteers throughout the U.S. and our six international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D.
To accomplish this, JDRF has invested more than $2.2 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact, and uniting on a national stage to pool resources, passion and energy. We collaborate with academic institutions, policymakers, and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D.
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