EMA has recommended granting a marketing authorisation in the European Union (EU) for Teizeild (teplizumab) to delay the onset of stage 3 type 1 diabetes in adults and in children from 8 years of age with stage 2 type 1 diabetes.
Type 1 diabetes is a chronic autoimmune disease where the body's immune system destroys beta cells in the pancreas that produce insulin, a hormone that regulates blood glucose (sugar) by allowing it to move into cells to produce energy. As a result, glucose builds up in the blood and causes multiple symptoms, like thirst, hunger, frequent urination, weight loss and tiredness. Over time, it can affect major organs in the body, including the heart, blood vessels, nerves, eyes and kidneys. Patients need daily insulin injections to control their glucose levels.
Type 1 diabetes often begins in childhood but can occur at any age. The disease progresses in three stages, and symptoms normally appear at stage 3, when daily insulin injections to manage blood glucose levels are required. This causes a significant burden to patients, especially children and their caregivers. Delaying the onset of stage 3 particularly in children is beneficial in diabetic care. An estimated 2.2 million people in the EU live with type 1 diabetes. There are currently no authorised treatments to delay or cure the disease.
The active substance of Teizeild is teplizumab, an antibody that delays disease progression by reducing the pace of the patient’s self-destruction of pancreatic beta cells. It is administered by intravenous infusion once daily for 14 consecutive days.
Teizeild was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs.
The recommendation is based on the results of a randomised, double-blind, placebo-controlled clinical trial in 76 patients with stage 2 type 1 diabetes. The median time to develop type 1 diabetes stage 3 was 50 months in patients treated with teplizumab and 25 months in patients who received placebo. Twenty (45%) of the 44 patients treated with teplizumab developed type 1 diabetes compared to 23 (72%) of the 32 patients who received placebo during the study (median follow up of 51 months). In addition, results from several other studies show that treatment with teplizumab significantly preserved the functioning of pancreatic beta cells compared to treatment with placebo.
The most common side effects reported with Teizeild were low levels of various types of white blood cells (lymphocytes, leucocytes and neutrophils), rash and low levels of blood bicarbonate, which can cause metabolic acidosis (too much acid in the body). The most frequent serious adverse reaction reported in 2% of patients was cytokine release syndrome, a serious condition causing fever, vomiting, shortness of breath, headache and low blood pressure. The product information and risk management plan of Teizeild include appropriate risk mitigating measures.
The opinion adopted by the CHMP is an intermediary step on Teizeild’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.
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