Sanofi announces that Teplizumab, a humanised anti-CD3 monoclonal antibody — the first of an emerging class of disease-modifying treatments — has been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA).
It has been licenced to delay the onset of symptomatic stage 3 autoimmune type 1 diabetes (T1D) in adult and paediatric patients 8 years of age and older with stage 2 T1D.
Following authorisation in the US, the UK is the first country in Europe to be granted a licence. Until now, available options have focused solely on managing the symptomatic disease.
It is known that T1D is not caused by diet or lifestyle, but the causes of T1D are currently not fully understood.
T1D develops gradually in three stages with time. Long before symptoms appear, the immune system mistakenly attacks the insulin-producing cells in the pancreas. By stage 3, most of the insulin-producing cells are destroyed and people become symptomatic.
From stage 3, people experience symptoms like increased thirst and weight loss; this is usually when they are diagnosed.
Once diagnosed, they face a daily regimen of multiple insulin injections or pump use, continuous glucose monitoring, strict dietary management and the risk of acute complications, including diabetic ketoacidosis, a potentially life-threatening condition.
Beyond the immediate physical demands, T1D carries a psychological toll, impacting mental health, social activities and overall quality of life.
The long-term complications of the disease include kidney disease, nerve damage, blindness and cardiovascular issues.
Teplizumab has been the subject of multiple clinical studies involving more than 1,000 subjects with more than 800 patients receiving teplizumab.
Parth Narendran, Professor of Diabetes Medicine, University of Birmingham, and The Queen Elizabeth Hospital Birmingham, commented: “Teplizumab trains the immune system to stop attacking the beta cells in the pancreas, allowing the pancreas to produce insulin without interference."
"This can allow eligible patients to live normal lives, delaying the need for insulin injections and the full weight of the disease's daily management by up to three years. It allows people to prepare for disease progression rather than facing an abrupt emergency presentation.”
Karen Addington, MBE, Chief Executive, Breakthrough T1D UK, added: “After years of research supported by Breakthrough T1D, I am delighted by the MHRA’s authorisation of teplizumab."
"We now have an immunotherapy that can delay the development of symptomatic type 1 diabetes (T1D) and may help families avoid life-threatening complications, such as diabetic ketoacidosis (DKA)."
"What teplizumab offers is time free from the daily challenges of T1D management, a potential reduced risk of serious complications, and the possibility of an improved quality of life."
Ahmed Moussa, General Manager, General Medicines UK and Ireland, Sanofi, concluded: "One hundred years ago, the discovery of insulin revolutionised diabetes care."
"Today’s news marks a big step forward. We are now working with reimbursement bodies with the hope of securing timely access for eligible patients across England and Wales.”