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The anti-CD3 antibody teplizumab (Tzield) has become the first drug indicated to delay the progression of type 1 diabetes, on Thursday.

Targeting the underlying autoimmune process of type 1 diabetes instead of just providing metabolic control, teplizumab is approved for adults and pediatric patients 8 years and older with stage 2 type 1 diabetes who want to delay the onset of stage 3, the irreversible condition when insulin-producing cells lose the ability to maintain normal glycemic control.

"Today's approval of a first-in-class therapy adds an important new treatment option for certain at-risk patients," said John Sharretts, MD, 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."

The biologic drug is to be administered by IV infusion once daily for 14 consecutive days. Each vial will cost $13,300 wholesale, bringing the price tag of full treatment to $193,900, teplizumab-maker Provention Bio during a conference call on Friday.

FDA approval comes more than a year after teplizumab's lukewarm reception at an FDA Endocrinologic and Metabolic Drugs Advisory Committee meeting in which advisors looked past the shortcomings of the limited evidence -- namely, the small, randomized TrialNet-10 trial -- to vote 10-7 that the biologic's benefits do outweigh its risks.

The trial included 76 patients with stage 2 type 1 diabetes and showed that a 14-day course of treatment significantly reduced the risk of of developing stage 3 disease. Over a median follow-up of 51 months, 45% of patients who received teplizumab were later diagnosed with stage 3 type 1 diabetes, compared with 72% of those who received a placebo.

Against the recommendation of many of those FDA advisors, the agency is not requiring a risk evaluation and mitigation strategy program for teplizumab.

In TrialNet-10, the most common side effects of teplizumab were lymphopenia, rash, leukopenia, and headache.

The also includes a warning of cytokine release syndrome as a potential side effect. Users are advised to premedicate and treat symptoms with antipyretics, antihistamines, and/or antiemetics. In severe cases, patients may pause dosing for 1 or 2 days and resume treatment; they should discontinue treatment entirely if liver enzymes cross dangerous thresholds during treatment.

Teplizumab is believed to work by deactivating the immune cells that attack insulin-producing cells, while increasing the proportion of cells that help moderate the immune response.

"It cannot be emphasized enough how precious a delay in the onset of stage 3 [type 1 diabetes] can be from a patient and family perspective; more time to live without and, when necessary, prepare for the burdens, complications and risks associated with stage 3 disease," said Ashleigh Palmer, co-founder and CEO of Provention Bio, in the drug manufacturer's .

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