With Sanofi as its marketing partner, Provention Bio grabs long-awaited FDA nod for diabetes drug

What a long, strange trip it’s been for teplizumab, a drug developed to combat juvenile diabetes—36 years to be exact.

On Thursday, when the FDA approved Provention Bio’s Tzield, it culminated a journey that began in 1986, the same year as Chernobyl, the Space Shuttle disaster and the debut of the Oprah Winfrey Show.

The immunotherapy, which delays the progression of type 1 diabetes, will be available to patients age 8 and older who are in stage 2 of the disorder. It becomes the first drug approved to delay progression of type 1 diabetes.

In testing, patients on Tzield progressed from stage 2 to stage 3 an average of 25 months later than those on placebo. Reaching stage 3 can present significant health risks, including the potentially fatal complication ketoacidosis.

“Today's FDA decision gives people at risk of developing type 1 diabetes the gift of time,” Aaron Kowalski, Ph.D., the CEO of the Juvenile Diabetes Research Foundation, said in a release. “For the first time ever, we have a way to change the course and slow the development of T1D. This is a huge win for the T1D community.”

There are roughly 30,000 patients in the U.S. who will become eligible for the treatment. But there is potentially a much larger population of undiagnosed patients. It is estimated that there are 1.4 million in the U.S. with stage 2 type 1 diabetes, Provention said.

The infused treatment will carry a list price of $13,850 per vial, which works out to $193,900 for a 14-vial continuous regimen for the average-sized patient, Jason Hoitt, Provention’s chief commercial officer, said in a conference call.

The price surprised analysts, such as Thomas Smith of SVB Securities, who expected a figure of $115,000, he wrote in a note to investors.

“This is not another symptomatic monoclonal antibody that’s treating symptoms better than the previous one,” said Provention CEO and founder Ashleigh Palmer, during the call. “This is a game-changing breakthrough innovation that brings a therapy to stage 2 type 1 diabetes patients that previously had no option other than wait for the progression of the disease.”

Last month, Provention revealed that it had struck a deal with Sanofi to market the drug in the United States. Sanofi will pay $35 million to Provention and an additional $20 million to secure the right to first negotiation on exclusive global marketing rights when other governments approve Tzield.

Under the deal, Sanofi will allow Provention call on its diabetes field specialists and account directors, plus field-based reimbursement and medical science liaisons, which should help Provention fill out its Rolodex of local healthcare professionals in the field.

The approval has special significance for the University of California San Francisco. It was there that immunologist Jeff Bluestone, Ph.D., kicked off the research leading to the development of teplizumab.

The drug made the rounds, from MacroGenics to Eli Lilly in 2007 and then to Provention in 2011, after it flunked a late-stage trial.

At Provention, it sat on the shelf until, at Bluestone’s urging, the New Jersey-based company gave it another shot based on lessons learned in the first go-round. Bluestone eventually joined the board at Provention in 2019.