FDA Approves Teplizumab to Delay the Onset of Type 1 Diabetes (2022)

On November 17, 2022, the U.S. Food and Drug Administration (FDA) approved teplizumab-mzwv, a medicine given as a 14-day course of daily intravenous infusions, to delay the onset of stage 3 type 1 diabetes in children and adults aged 8 and older who are at high risk of the disease. The approval was based on one placebo-controlled trial of 76 people, in which those given the medicine were diagnosed with type 1 diabetes later, and less often during follow-up, than those given placebo. The disease itself is described on the site’s Type 1 Diabetes page.

This page reports what the FDA’s own documents say: what was approved and when, who counts as “high risk,” how the medicine is given, the trial behind the approval and its results, the side effects the FDA lists, what the approval does not do, its status as of October 18, 2026, and how it fits other FDA drug actions of 2022. No brand, manufacturer or trial code is named here.

Table of Contents

  1. What the FDA Approved on November 17, 2022
  2. Stages of Type 1 Diabetes and Who Counts as High Risk
  3. How the Medicine Is Given
  4. The Trial Behind the Approval
  5. What the Trial Found
  6. Differences by Sex, Age and Race
  7. Side Effects the FDA Lists
  8. What the Approval Does Not Do
  9. Dates and Legal Status as of October 18, 2026
  10. How It Fits Other FDA Drug Actions of 2022
  11. Primary Documents
  12. Key Research Papers
  13. Connections
  14. Featured Videos

1. What the FDA Approved on November 17, 2022

The FDA’s Drug Trials Snapshot for the medicine gives its “Original Approval date” as November 17, 2022, and states that it is “a drug that is used to delay the onset of type 1 diabetes in children and adults who are at high risk for type 1 diabetes.” The FDA’s list of Novel Drug Approvals for 2022 records the approved use as “To delay the onset of stage 3 type 1 diabetes.”

A Drug Trials Snapshot is a short FDA summary, written for the public, of who took part in the key clinical trials behind a drug’s original approval and whether results differed by sex, race, age or ethnicity. The FDA’s Novel Drug Approvals list records the new medicines its Center for Drug Evaluation and Research approved in a year; teplizumab-mzwv is entry 30 on the 2022 list, which runs to 37 entries.

The Snapshot describes type 1 diabetes in these words: “a lifelong condition where your body cannot make enough insulin on its own to keep blood sugar levels normal, and you need daily insulin injections or an insulin pump.” It describes the medicine as “a prescription medicine that delays the onset of type 1 diabetes in patients who are at high risk for having the disease.”

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2. Stages of Type 1 Diabetes and Who Counts as High Risk

The approval is for people who do not yet have a diagnosis of type 1 diabetes but whose blood tests show the disease process has started. The Snapshot explains: “A high-risk person has two or more type 1 diabetes-related autoantibodies and abnormal blood sugar levels that are not yet high enough for a diagnosis of diabetes.”

An autoantibody is an antibody the immune system makes against the body’s own tissue. The trial behind the approval enrolled people with what it called stage 2 type 1 diabetes, which, according to the Snapshot, meant having both of the following:

  1. Two or more of these pancreatic islet autoantibodies (the islets are the clusters of cells in the pancreas that make insulin):
    • glutamic acid decarboxylase 65 (GAD) autoantibodies;
    • insulin autoantibody (IAA);
    • insulinoma-associated antigen 2 autoantibody (IA-2A);
    • zinc transporter 8 autoantibody (ZnT8A);
    • islet cell autoantibody (ICA).
  2. Dysglycemia (abnormal blood sugar) on an oral glucose tolerance test, a test in which blood sugar is measured after a sugary drink.

The point the trial measured was the move from stage 2 to stage 3, which the Snapshot treats as the diagnosis of type 1 diabetes: the trial’s primary endpoint was “the time from randomization to development of stage 3 type 1 diabetes diagnosis.” The Snapshot also notes that “People who are at high risk for type 1 diabetes are generally children and younger adults.”

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3. How the Medicine Is Given

According to the Snapshot, the medicine “is an injection given by a healthcare professional directly into a vein (intravenous infusion) once per day for 14 days.” The course described in the Snapshot is one series of daily infusions lasting 14 days, each given by a health care professional.

The Snapshot carries its own limitation: “Do not rely on Snapshots to make decisions regarding medical care,” and it points readers to the full prescribing information “for all of the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).” Decisions about any diabetes medicine belong with a clinician.

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4. The Trial Behind the Approval

The Snapshot states that the FDA approved the medicine “based on evidence from one clinical trial” of 76 patients at high risk of developing type 1 diabetes. The trial was run at 30 sites in five countries: the United States, Canada, Australia, the United Kingdom and Germany.

The trial was randomized (participants were assigned to a group by chance), double-blind (neither participants nor investigators knew who received which) and placebo-controlled. It enrolled people aged 8 to 49 with stage 2 type 1 diabetes, who received either the medicine or placebo once a day by intravenous infusion for 14 days. A placebo, in the Snapshot’s glossary, is “An inactive substance or ‘sugar pill’ that looks the same as, and is given the same way as, an active drug or treatment being tested.”

Who took part, according to the Snapshot’s demographics table: 44 people received the medicine and 32 received placebo. In the medicine group, 43% were female and 66% were under 18; in the placebo group, 47% were female and 81% were under 18. All 44 participants in the medicine group were White; in the placebo group, 30 of 32 were White, one was Asian and one was of multiple races. Two participants in total were Hispanic or Latino.

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5. What the Trial Found

The Snapshot summarizes the benefit in plain language: during the trial, which followed each participant for about 51 months, those taking the medicine had a 59% lower risk of developing type 1 diabetes than those taking placebo. It adds that participants on the medicine “developed type 1 diabetes generally 50 months after receiving the medication, compared to 25 months among those taking placebo.”

The Snapshot’s Table 2, adapted from the FDA review, gives the figures behind those statements:

A hazard ratio compares how often an event happens over time in one group against another; 0.41 means the rate of diagnosis in the medicine group was about 41% of the rate in the placebo group, which is the “59% lower risk” in the Snapshot’s summary. A median is the middle value: half the participants were diagnosed before that time and half after. The 95% confidence interval is the range within which the true value is likely to lie, given the size of the trial.

The result is a delay. During the trial, diagnoses still occurred in both groups, including in almost half of the people who received the medicine.

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6. Differences by Sex, Age and Race

The Snapshot reports that the medicine “worked similarly in males and females” and “worked similarly in all age groups.” On race, it states that the number of participants of races other than White was small, so differences in how the medicine worked among races “could not be determined.”

Its Table 3 gives subgroup hazard ratios, with the caution that the estimates draw on results from the other subgroups: 0.22 for females (34 participants) and 0.58 for males (42); 0.37 for ages 8 to under 11 (18 participants), 0.34 for 11 to under 14 (19), 0.62 for 14 to under 18 (18) and 0.41 for 18 and older (21). Each subgroup was small, and several of the intervals around these numbers are wide.

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7. Side Effects the FDA Lists

The Snapshot lists three serious side effects:

The most common side effects, according to the Snapshot, were lymphopenia (“decrease in a certain type of white blood cells called lymphocytes”), rash, leukopenia (“decrease in the total number of white blood cells”) and headache. Its Table 4, taken from the prescribing information, gives how often these occurred in the 76-person trial, medicine group versus placebo group:

The wider safety pool. Beyond the main trial, the Snapshot states that safety was evaluated in four more controlled trials in a population for which the medicine is not approved. In total, 773 people received the medicine and 245 received placebo or a control treatment. Of the 773, 436 received a 14-day course with total exposure comparable to the approved dose, 168 a 14-day course with lower exposure and 169 a 6-day course with lower exposure. In that pooled analysis, limited to the 14-day treatment period, serious adverse events were reported in 10 of 773 people on the medicine and 1 of 245 on control.

The Snapshot reports that side effects were similar in males and females and showed no differences by age or race, although safety data for races other than White and Asian were limited, and that “Studies to assess side effects generally did not include those 65 years of age and older.”

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8. What the Approval Does Not Do

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9. Dates and Legal Status as of October 18, 2026

Status as of October 18, 2026: approval in effect. A query of the Federal Register, the U.S. government’s daily publication of rules and official notices, for “teplizumab” from January 1, 2022 through October 18, 2026 returned no documents, so no withdrawal, suspension or other change to the approval has been published there. The approval is recorded in the FDA documents listed in section 11.

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10. How It Fits Other FDA Drug Actions of 2022

The FDA’s Center for Drug Evaluation and Research lists 37 novel drug approvals for 2022. This one is approved to delay the diagnosis of a disease in people who do not yet meet its diagnostic threshold, rather than to treat diagnosed disease. The immune system is covered in the site’s Immunology section.

Other FDA medicine approvals of 2022 are reported on their own pages: the May 2022 approval of tirzepatide for type 2 diabetes, the September 2022 ALS drug approval that was later withdrawn and the November 2022 approval of a fecal microbiota product for recurrent C. difficile infection. The full list of 2022 actions is on the FDA Actions of 2022 hub.

The disease itself is described on the site’s Type 1 Diabetes and Diabetes pages.

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11. Primary Documents

  1. U.S. Food and Drug Administration. Drug Trials Snapshot for teplizumab-mzwv (delay of onset of type 1 diabetes), original approval November 17, 2022. Content current as of October 10, 2024. Listed among the FDA’s Drug Trials Snapshots on fda.gov; its web address carries the product’s brand name, so it is given here as plain text without a link.
  2. U.S. Food and Drug Administration. Novel Drug Approvals for 2022 (entry 30, teplizumab-mzwv, “To delay the onset of stage 3 type 1 diabetes”). Content current as of January 2, 2025 — fda.gov Novel Drug Approvals for 2022
  3. Federal Register document query for “teplizumab,” publication dates January 1, 2022 to October 18, 2026 (no documents returned), run through the federalregister.gov public API on October 18, 2026.

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Key Research Papers

  1. Herold KC, Bundy BN, Long SA, Bluestone JA, DiMeglio LA, Dufort MJ, Gitelman SE, Gottlieb PA, Krischer JP, Linsley PS, Marks JB, Moore W, Moran A, Rodriguez H, Russell WE, Schatz D, Skyler JS, Tsalikian E, Wherrett DK, Ziegler AG, Greenbaum CJ; Type 1 Diabetes TrialNet Study Group (2019). An Anti-CD3 Antibody, Teplizumab, in Relatives at Risk for Type 1 Diabetes. New England Journal of Medicine 381(7):603-613 — PubMed PMID: 31180194
  2. Sims EK, Bundy BN, Stier K, Serti E, Lim N, Long SA, Geyer SM, Moran A, Greenbaum CJ, Evans-Molina C, Herold KC; Type 1 Diabetes TrialNet Study Group (2021). Teplizumab improves and stabilizes beta cell function in antibody-positive high-risk individuals. Science Translational Medicine 13(583):eabc8980 — PubMed PMID: 33658358
  3. Insel RA, Dunne JL, Atkinson MA, Chiang JL, Dabelea D, Gottlieb PA, Greenbaum CJ, Herold KC, Krischer JP, Lernmark Å, Ratner RE, Rewers MJ, Schatz DA, Skyler JS, Sosenko JM, Ziegler AG (2015). Staging presymptomatic type 1 diabetes: a scientific statement of JDRF, the Endocrine Society, and the American Diabetes Association. Diabetes Care 38(10):1964-1974 — PubMed PMID: 26404926
  4. Herold KC, Hagopian W, Auger JA, Poumian-Ruiz E, Taylor L, Donaldson D, Gitelman SE, Harlan DM, Xu D, Zivin RA, Bluestone JA (2002). Anti-CD3 monoclonal antibody in new-onset type 1 diabetes mellitus. New England Journal of Medicine 346(22):1692-1698 — PubMed PMID: 12037148
  5. Ramos EL, Dayan CM, Chatenoud L, Sumnik Z, Simmons KM, Szypowska A, Gitelman SE, Knecht LA, Niemoeller E, Tian W, Herold KC; PROTECT Study Investigators (2023). Teplizumab and β-Cell Function in Newly Diagnosed Type 1 Diabetes. New England Journal of Medicine 389(23):2151-2161 — PubMed PMID: 37861217

The first paper is the published report of a randomized trial of teplizumab in relatives of people with type 1 diabetes who were at high risk of the disease, and the second reports measures of insulin-producing (beta) cell function in antibody-positive high-risk individuals given teplizumab. The third is the scientific statement that set out the staging of presymptomatic type 1 diabetes. The fourth is an early trial of the same class of antibody in people newly diagnosed with type 1 diabetes, and the fifth a later trial of teplizumab in people newly diagnosed with type 1 diabetes, a use outside the 2022 approval.

PubMed Topic Searches

  1. PubMed: teplizumab
  2. PubMed: presymptomatic type 1 diabetes staging
  3. PubMed: islet autoantibody screening for type 1 diabetes

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Connections

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