By JOE DAVIES HEALTH REPORTER FOR MAILONLINE
PUBLISHED: 03:09 EST, 18 November 2022 | UPDATED: 07:50 EST, 18 November 2022
A world-first drug that can delay the later stages of type 1 diabetes by up to three years was today hailed as a ‘seismic shift’ in the race to beat the illness.
Regulators at the US Food and Drug Administration (FDA) approved teplizumab for patients aged eight and above.
Doctors in the US described it as a ‘watershed moment’ for the treatment and prevention of diabetes.
British charities said the drugs approval was a ‘milestone’ in treating the condition and called for it to be given the green light in the UK ‘as soon as possible’ – with UK officials still assessing the drug.
Teplizumab — sold as Tzield — is a type of immunotherapy which works by tackling the root cause of type 1 diabetes rather than just its symptoms.
The drug tells the body not to attack pancreatic cells which produce insulin. These are vital for controlling blood glucose levels.
Protecting these cells can help buy people in the early stages of the condition more time before they become dependent on taking insulin shots.
In the US, it is approved for patients in stage two type 1 diabetes — when they have abnormal blood sugar levels but their bodies still make insulin.
They take a daily infusion lasting up to an hour for two weeks that can help stave off more serious symptoms at stage three, when it is clinically diagnosed.
Teplizumab — sold as Tzield — is a type of immunotherapy that works by tackling the root cause of type 1 diabetes rather than just its symptoms. It is currently being reviewed for use in the UK
Type 1 diabetes affects about 400,000 people in the UK, including more than 29,000 children. Graph shows: The age of diagnosis for people with type 1 diabetes in England and Wales from 2019-20
In stage three type 1 diabetes, the pancreas no longer makes any insulin, so people have to inject it to keep their blood sugar levels steady.
The auto-immune condition affects about 400,000 people in the UK, including more than 29,000 children. Some 1.45million Americans live with it.
Unlike type 2 diabetes, it is not caused by being fat.
Experts are still baffled as to exactly what triggers it, but genetics are thought to be to blame.
Around 85 per cent of people diagnosed with type 1 diabetes have no family history of the condition.
Symptoms, such as feeling very thirsty, passing urine more than usual, losing weight without trying and being very tired, can come on quickly, particularly in children.
If left untreated, type 1 diabetes is life-threatening.
Dr Mark S. Anderson, director of the University of California San Francisco Diabetes Center, told ABC News: ‘This approval is a watershed moment for the treatment and prevention of type 1 diabetes.’
Chris Askew, chief executive of Diabetes UK, said: ‘Today’s landmark approval of teplizumab in the US is the start of a seismic shift in how type 1 diabetes is treated.
‘For 100 years, people living with type 1 diabetes have relied on insulin to treat the condition, and today’s decision means that for the first time, the root cause of the condition — an immune system attack — can be tackled and type 1 diabetes potentially delayed for up to three years.
‘The licensing of teplizumab in the UK must now be accelerated, and we’re working with the NHS and with other diabetes charities and key stakeholders to ensure that people in the UK can benefit from this life-changing treatment as soon as possible.’
Mr Askew said preventative treatments for type 1 diabetes must be combined with screening programmes to identify people at risk.
He added: ‘Diabetes UK is funding immunotherapy research to help people at all stages of type 1 diabetes, and we hope this monumental breakthrough will open the door for increased research investment, to develop further effective immunotherapies to treat the condition.
‘Today is a significant moment in the history of type 1 diabetes and one that will shape the future — propelling us closer to the day where type 1 diabetes can be prevented or cured altogether.”What is diabetes?’ Diabetes UK explains the condition
The JDRF charity, which funded a trial into the drug, said teplizumab also has the potential to slow progression of the illness in the long term.
Karen Addington, chief executive of JDRF UK, said: ‘The world now has a drug which is proven to effectively tackle the root causes of type 1 diabetes, delaying the onset of the condition and slowing down disease progression.
‘Our work now focuses on securing approval in the UK. We will also be leading further research into the future potential of teplizumab to entirely prevent type 1, helping to eradicate this condition from everyone’s lives.’
The Medicines and Healthcare products Regulatory Agency (MHRA) — which decides what drugs are approved in the UK — told MailOnline: ‘Once we receive any application for marketing authorisation, we review it as quickly as possible without compromising our standards on safety, quality and effectiveness.’
Reacting to the FDA’s approval, Ashleigh Palmer, the co-founder of Provention Bio which makes Tzield, said the drug works by ‘fundamentally changing the course of the disease’.
He said: ‘This is a historic occasion for the T1D community and a paradigm shifting breakthrough for individuals aged eight years and older with Stage 2 T1D who now have a therapy approved by the FDA to delay the onset of Stage 3 disease.
‘It cannot be emphasised 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.’
‘We expect to have drug in the channel by the end of the year,’ said Jason Hoitt, Provention’s chief commercial officer, adding that preparations were underway with its partner Sanofi for a full launch in January 2023.
In October, Provention signed a co-promotion deal for the drug with Sanofi, offering the French drugmaker first negotiation for exclusive global rights to commercialize the drug in exchange for an upfront payment of $20million (£16.7million).
‘We expect that there is a greater value of teplizumab beyond this indication and hope for a broader collaboration with Provention in the future,’ said Olivier Bogillot, Sanofi’s head of U.S. General Medicines.
As per the deal, the approval will also allow Sanofi to purchase up to $35million (£29.3million) of Provention’s common stock.
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