A medical breakthrough gives hope to tens of thousands of people with Crohn’s disease.
A new daily tablet for the debilitating digestive system condition has received its first global approval in the UK.
Upadactinib is now set to become available on the NHS for the 200,000 people whose lives are being destroyed by its painful and embarrassing symptoms.
Once given full power, the pill can be prescribed instead of regular injections or infusions or steroids.
It works by turning off signals in the body that cause inflammation in the stomach and intestines – the source of the disease’s hallmark symptoms.
Student Sinead Burnham, 20, pictured above, was diagnosed with Crohn’s when she was 15 years old. She now receives bi-weekly injections to control the condition and believes the availability of a once-a-day pill will help some people with Crohn’s disease
The pill, pictured above, could be prescribed instead of regular injections or infusions or steroids once it has full authority, works by turning off signals in the body that cause inflammation in the stomach and intestines and trials have shown it can be effective occur in up to 60 percent of patients
Trials have shown that upadactinib can be effective in up to 60 percent of patients, putting some with moderate to severe disease into long-term remission.
It is approved by the Medicines and Healthcare products Regulatory Agency (MHRA), which oversees the safety of medicines in the UK.
And it has just been recommended by the European Medical Agency, bringing it one step closer to availability on the NHS.
Crohn’s disease, an incurable form of inflammatory bowel disease, normally affects people in their teens and early 20s or mid-50s.
The disease, which affects comedian Pete Davidson and magician Dynamo, causes significant physical, emotional and economic burdens, with patients having to go to the toilet frequently and struggling with work and relationships.
Other symptoms include crippling stomach pain, diarrhea, swollen joints, fatigue, and depression.
Professor James Lindsay, gastroenterologist consultant at the Royal London Hospital Barts Health NHS Trust, said: ‘Limited new treatment options have been approved for Crohn’s disease in recent years and many people are struggling to stay in remission.
“This is not a cure, but it has the potential to help people manage their disease.
“Patients want predictability, to know they will be fine for a while so they can make plans to return to normal life.
‘Crohn’s disease has a very high burden of care, both in terms of direct healthcare costs, medicines, hospital admissions, and indirectly in terms of work productivity, absenteeism and inability to live your life as expected.
‘Getting people better is very good for those costs, but also for the physical and emotional gain for patients.’
The drug is a JAK inhibitor, which is already approved for patients with ulcerative colitis – a similar condition.
It works by blocking the rogue signals that drive an immune response to attack normal gut bacteria, which are at the heart of the inflammation caused by Crohn’s disease.
The MHRA approved the drug for patients who failed or responded inadequately to conventional or biologic therapies after clinical trials showed long-term remission of symptoms.
It is currently under review by NICE, the drug watchdog that approves drugs for use by the NHS, with a decision expected by the summer.
Sarah Sleet, managing director of the charity Crohn’s & Colitis UK, said: ‘There are over 200,000 people living with Crohn’s in the UK.
“It’s a lifelong condition that causes painful, debilitating symptoms and there’s no known cure.
“For some people with Crohn’s disease, existing treatments may not work, or they may stop working over time. Therefore, we welcome the MHRA’s decision to approve upadacitinib.
“This is a promising step forward in expanding treatment options for people with this condition.”
Roopal Thakkar, AbbVie senior vice president, development, regulatory affairs and chief medical officer, said: “The recent recommendation to approve upadacitinib for use in Crohn’s disease is an important step that moves us closer to offering a first of its kind, once-daily oral treatment that can make a difference for people with this disease.”