Red seal and imprint "FDA APPROVED" on white surface. FDA - Food and Drug Administration is a federal agency of the United States Department of Health and Human Services.

Red stamp and imprint "FDA APPROVED" on a white surface.  FDA - The Food and Drug Administration is a federal agency within the United States Department of Health and Human Services.

Patients with type 1 diabetes whose disease has reached its most advanced stage require lifelong insulin therapy to protect against potentially life-threatening complications. Prevention Bio medicine received FDA approval as a first treatment that delays the onset of this stage of the disease.

The approval of Provention’s drug, teplizumab, covers adults as well as children 8 years and older whose type 1 diabetes (T1D) is classified as stage 2. Red Bank, New Jersey-based Provention will market its new product under the brand name “Tzield.”

Type 1 diabetes is an autoimmune disease in which the immune system attacks the insulin-producing beta cells of the pancreas. The decline in beta cell function is classified according to three stages. By the third stage, when most patients are diagnosed, a significant proportion of the beta cells have already been lost. These patients usually need regular insulin injections for life to manage their blood sugar levels.

Tzield is an antibody designed to bind to CD3, a receptor on T cells that activates them and directs the immune response. Binding to this receptor is intended to deactivate T cells, preventing them from attacking beta cells. This approach also aims to increase the proportion of regulatory T cells, a different type of immune cell that suppresses the immune response. Although Tzield does not stop type 1 diabetes, the drug aims to slow the progression of the disease.

The FDA’s decision, announced late Thursday, was based on the results of a placebo-controlled trial that evaluated 76 patients whose type 1 diabetes was classified as stage 2. Trial participants were randomly assigned to receive Tzield or a placebo administered as intravenous infusion once a day for 14 days. The main goal of the study is to measure the time it takes for a patient to develop stage 3 disease.

At a median follow-up of 51 months, 45 percent of the 44 patients who received the Provention drug were later diagnosed with stage 3 diabetes, compared with 72 percent of the 32 participants who received a placebo. The median onset of stage 3 diabetes was 25 months for the Tzield group compared with 50 months for the placebo group. The results were sufficient to show a statistically significant delay in the development of stage 3 type 1 diabetes. The study data were published last year in the New England Journal of Medicine.

“The resulting median delay of more than two years in the onset of stage 3 disease is an invaluable therapeutic outcome for patients with stage 2 T1D,” Provention CEO Ashley Palmer said on a conference call Friday.

Of course, Tzield comes at a price. Provention set a wholesale price of $13,850 per vial. This means that a 14-day dosing regimen will cost $193,900. Dosing is based on patient weight, and some patients may require additional vials. Provention will commercialize Tzield under a partnership agreement reached with Sanofi last month. Under the agreement, Provention retains the rights to Tzield, but will employ Sanofi’s diabetes specialists and other personnel. Provention will reimburse Sanofi up to $33 million in fiscal 2023.

Sanofi has committed $55 million to the alliance, which amounts to a non-refundable payment of $20 million for the right to first negotiate for global rights to Tzield in type 1 diabetes. That right to negotiate expires in June 2023, although it can be extended under conditions not specified. In addition, Sanofi agreed to buy $35 million worth of Provention shares at a 40% premium by February 16.

The most common side effects reported in clinical trials were a decrease in the levels of certain types of white blood cells, rash and headache. The drug’s label states the potential risk of cytokine release syndrome, an excessive immune response that has been reported in 5% of patients given Tzield. This side effect can be mitigated by pre-treating the patient and monitoring for signs and symptoms.

Last year, an FDA advisory committee voted 10-7 on whether the benefits of the drug Prevention outweigh the risks. Palmer said that as a condition of Tzield’s approval, Provention has committed to creating a registry to monitor long-term safety. The company will also conduct a pediatric study in patients with type 1 diabetes under the age of 8 whose disease has reached stage 2.

Additional studies are underway that could potentially support expanding the drug’s use to more patients with type 1 diabetes. A phase 3 study in newly diagnosed patients is fully enrolled; preliminary data is expected in the second half of next year, according to an investor presentation. Additional potential expansion options include treating children between the ages of 2 and 8, re-dosing the therapy and a subcutaneous injection version of the drug. Provention is also investigating the drug in autoimmune conditions that include early rheumatoid arthritis, celiac disease, Crohn’s disease and autoimmune hepatitis.

Provention licensed Tzield by Macrogenics. With FDA approval of the drug, Provention will pay the Rockville, Maryland-based biotech a Key payment of $60 million.

Photo: Waldemarus, Getty Images

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