FDA Approves Tzield (Teplizumab) to Delay Type 1 Diabetes
By Arvind SommiEugenia Yun
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The FDA has approved Provention Bio’s Tzield (teplizumab), a new therapy that could delay the onset of type 1 diabetes by an average of 2 years. Find out about screening and how to know whether treatment with Tzield may be right for you.
Finding a cure for type 1 diabetes (T1D) has been a priority in diabetes research for decades. Today’s FDA approval of Tzield brings us potentially one step closer to that cure by providing a way to delay the onset of T1D, and marks an impressive milestone in diabetes research.
Tzield, developed by Provention Bio, is the first and only treatment to delay onset of stage 3 T1D in adults and children aged 8 and older with stage 2 T1D. It is an injectable drug taken once daily for 14 days that binds to and modifies cells in the immune system called T-cells. In people with T1D, T-cells destroy the insulin-producing beta cells in the pancreas. With the help of Tzield, this self-destructive process is interrupted, effectively delaying the onset of T1D by an average of about 2 years.
"This is a historic occasion for the T1D community and a paradigm-shifting breakthrough for individuals aged 8 years and older with stage 2 T1D who now have a therapy approved by the FDA to delay the onset of stage 3 disease,” said Ashleigh Palmer, co-founder and CEO of Provention Bio in a statement.
“It cannot be emphasized enough how precious a delay in the onset of stage 3 T1D 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,” he said.
Who is eligible to receive Tzield?
Tzield is approved for people who are at high risk for T1D and have abnormal blood sugar levels determined by an oral glucose tolerance test. To meet the criteria for “high risk,” a person must have a family history of T1D and a screening test that confirms the presence of at least two specific autoantibodies that indicate that the body is already attacking the insulin-producing beta cells in the pancreas.
There are three stages of type 1 diabetes:
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Stage 1. Blood sugar levels are normal and the person experiences no symptoms of T1D, but the person has two or more autoantibodies associated with T1D. People who test positive for at least two autoantibodies are almost certain to develop T1D at some point in their lives.
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Stage 2. There are no symptoms of T1D, but blood sugar levels are not normal, and the autoantibodies for T1D persist.
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Stage 3. Blood sugar levels are high, and the person typically experiences symptoms of T1D (frequent urination, dry mouth, fatigue, and/or diabetic ketoacidosis). T1D is not usually diagnosed until stage 3.
Tzield is indicated for people with a family history of T1D who have stage 2 of T1D. Prior research has revealed that Tzield is not as effective in stage 3 T1D, once beta cells have already been destroyed.
Moreover, the drug is only approved for people ages 8 and older. This is a significant limitation since many people are diagnosed with T1D before age 8.
How to get tested for type 1 diabetes autoantibodies
Screening for T1D autoantibodies is extremely important for early detection and treatment of the condition. Although people had been reluctant to get screened in the past because there was not much that could be done, this new intervention might address the reason for this hesitation.
Here are three ways to get screened:
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At-home testing. This is a simple test that can be completed at home and mailed back. If the results are positive, a confirmatory test should be taken at a doctor’s office. This test is associated with JDRF’s T1Detect program. The cost of this test is $55, with reduced-cost options for anyone in financial need.
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TrialNet. This is a clinical trial program that provides free testing to those who have a family connection to T1D.
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Doctor’s office or lab. A doctor can order a blood test that may be done in the clinic or a lab, such as LabCorp or Quest Diagnostics.
People who test positive for two or more autoantibodies will be called to discuss results and develop a plan moving forward.
Provention Bio, the manufacturer of Tzield, has developed a resource guide about how to get screened for health care professionals and families with type 1 diabetes.
Potential risks
There are potential benefits to delaying T1D with Tzield, but there are also risks to taking it or any drug. The most common side effects included decreased levels of certain white blood cells, rash, and headache. The use of Tzield comes with warnings and precautions, including premedicating and monitoring for symptoms of cytokine release syndrome (when the immune system responds too aggressively to an infection or immunotherapy drugs); risk of serious infections; decreased levels of white blood cells; risk of hypersensitivity reactions; the need to administer all age-appropriate vaccinations before starting Tzield; as well as avoiding certain vaccines while using Tzield.
“We will be committed to setting up a registry to monitor Tzield’s long-term safety, as well as conducting a pediatric…safety study in stage 2 patients below 8 years of age,” said Palmer.
Most people in clinical trials who experienced side effects had mild symptoms that resolved within a few weeks.
How to access Tzield
Once a health care provider confirms eligibility for Tzield and decides with the patient that it is right for that person, they will fill out an enrollment form and send it to Provention Bio.
One of the company’s patient navigators from their COMPASS program will then reach out. The patient navigator will be the person’s guide and main point of contact through the rest of the process.
“COMPASS is our in-house patient support program with a staff of dedicated navigators available to answer questions and remove barriers for patients to access Tzield by helping them navigate coverage and reimbursement. This includes administration of our copay assistance program for eligible commercially insured patients to address financial barriers to treatment, including costs for both Tzield and its administration,” said Jason Hoitt, chief commercial officer at Provention Bio.
Tzield cost
The wholesale cost per vial of Tzield is $13,850, which translates to $193,900 for a 14-vial continuous regimen. Some people may require additional drugs beyond the 14 vials, depending on body surface area and weight.
Provention Bio offers patient assistance programs for those without insurance or who are unable to pay for Tzield. Once being prescribed Tzield, you and your healthcare provider can enroll in the COMPASS program. The program's patient navigators can help handle any insurance questions and financial assistance. Patients or health care providers can call COMPASS at 844-778-2246 or visit the Tzield website.
What’s next?
Tzield is currently being investigated in a phase 3 clinical trial of children and adolescents ages 8 through 17 within 6 weeks of stage 3 T1D diagnosis to determine if the drug may have benefit in that group.
“We will continue to explore and develop Tzield’s potential in younger patients [and] newly diagnosed patients, and investigate whether Tzield could be either re-dosed or combined with other treatments for even greater benefits,” said Hoitt.
To read more about our coverage of teplizumab, type 1 diabetes delay or prevention, and screening, read:
