The CD3 antigen inhibitor Tzield (teplizumab-mzwv) has won FDA approval to delay stage 3 type 1 diabetes in individuals at least 8 years old.
A vial of Tzield will have a wholesale cost of $13,850, announced the drug’s developer Provention Bio (Nasdaq:PRVB). The drug is administered as an intravenous infusion once daily for 14 consecutive days, resulting in an overall cost of $193,900.
Jason Hoitt
“How can one really value more time for a person — a child and their loved ones — without the psychological and physical impact that comes with stage 3 [type 1 diabetes] diagnosis?” asked Jason Hoitt, Provention Bio’s chief commercial officer, in a company presentation.
Hoitt said the FDA approval was “100 years in the making.” “The type 1 diabetes community has been waiting since the introduction of insulin for therapeutic advancement in the field.
Researchers at the University of Toronto first discovered insulin in 1921.
Provention Bio will partner with Sanofi (Nasdaq:SNY) to commercialize the drug. The French drugmaker agreed to pay a total of $55 million for marketing rights covering Tzield.
Treating type 1 and 2 diabetes is expensive. CDC estimates that $1 out of every $4 in U.S. health care costs is spent diabetes treatment.
Some patients in the U.S. ration insulin to save money.
Provention Bio has established a program known as Compass to ensure that uninsured patients have access to Tzield. “We want every person who may benefit from Tzield to have access to this important therapy,” Hoitt said.
The company acknowledges that some patients may require more than 14 vials. “But we believe that for the majority of patients, including those identified through familial screening and through general population screening and pediatrics, 14 vials will be sufficient,” Hoitt said.
In a press release, FDA said that the drug was an important new treatment option for some at-risk patients. “The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease,” said Dr. John Sharretts, director of the Division of Diabetes, Lipid Disorders and Obesity at the FDA’s Center for Drug Evaluation and Research (CDER), in a news release.
Tzield is the first disease-modifying therapy to win approval for type 1 diabetes.
The drug’s FDA indication covers individuals with stage 2 type 1 diabetes, which is presymptomatic but involves dysregulated autoantibodies and poor blood sugar control.
In afternoon trading, PRVB shares were down 0.55% to $8.20.
Many analysts had expected that the company would price the drug lower than it did.
Tzield is designed to deactivate immune cells that attack the beta cells that produce and secrete endogenous insulin.
To win FDA approval, the company provided data from a randomized, double-blinded, placebo-controlled study involving 76 patients with stage 2 type 1 diabetes. Fewer than half, 45%, of teplizumab developed stage 3 type diabetes. Conversely, 72% of placebo recipients did.
The drug will include labeling describing the possibility of cytokine release syndrome and other risks.
FDA had given the drug Priority Review and Breakthrough Therapy designations.