NICE RECOMMENDS STELARA® (USTEKINUMAB) FOR THE TREATMENT OF MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS
NICE RECOMMENDS STELARA® (USTEKINUMAB) FOR THE TREATMENT OF MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS
STELARA® (ustekinumab) is the first approved treatment to selectively target the
IL-12/IL-23 pathways, an important therapeutic target in ulcerative colitis
High Wycombe, UK, 28 April 2020 – The Janssen Pharmaceutical Companies of Johnson & Johnson welcomes the National Institute for Health and Care Excellence (NICE) positive Final Appraisal Document (FAD) recommending STELARA® (ustekinumab) as an option for treating moderately to severely active ulcerative colitis (UC) in adults when conventional therapy or a biological agent cannot be tolerated, or the disease has responded inadequately or lost response to treatment, only if:
- a tumour necrosis factor (TNF)-alpha inhibitor has failed (that is the disease has responded inadequately or has lost response to treatment)
- a TNF-alpha inhibitor cannot be tolerated or is not suitable.[i]
UC is a chronic immune-mediated inflammatory disease of the rectum and large intestine, affecting approximately 146,000 people in the UK, for which there is currently no cure.[ii] For up to one-third of people with UC, current treatments are not completely successful or complications may arise, where surgery may be considered.[iii]
“We are delighted that NICE revisited its decision and will now recommend ustekinumab for use in ulcerative colitis, as it is in Crohn’s disease. The impact of uncontrolled symptoms of ulcerative colitis can be devastating and debilitating. Current treatments simply do not work for everyone, so having as many treatment options as possible available, and allowing doctors and other clinicians to personalise their approach, is critical,” commented Sarah Berry, Health Service Project Manager, Crohn’s & Colitis UK*.
Ustekinumab is the first biologic therapy for UC that targets interleukin (IL)-12 and IL-23 cytokines, known to play a key role in inflammatory and immune responses,[iv] offering clinicians and patients a new mechanism of action for treating UC.[v]
“Ulcerative colitis can have a devastating impact on the lives of those living with this long-term condition. A flare up of symptoms can be incredibly debilitating, preventing people getting on with normal day-to-day life and work. Many patients do not respond to, or get side effects from, currently available therapies. Therefore, the recent NICE approval for ustekinumab as a novel therapy for patients that are not suited to currently available treatments is welcome,” commented Professor James Lindsay**, Consultant Gastroenterologist, Barts Health NHS Trust, London.
The NICE guidance is based on data from the pivotal Phase 3 UNIFI trial programme which evaluated ustekinumab’s safety and efficacy as a treatment option for adult patients with moderately to severely active UC. The programme was split into an initial Induction study (UNIFI-I) of eight weeks, followed by a Maintenance study (UNIFI-M) of 44 weeks. All patients who were randomised into the 44-week Maintenance study were induction responders to IV ustekinumab. Among patients subsequently randomised to receive subcutaneous ustekinumab (q8w or q12w), a significantly greater percentage achieved clinical remission at week 44 vs initial responders randomised to placebo (44 percent and 38 percent, vs 24 percent, p<0.001 and p=0.002, respectively).5
Jennifer Lee, EMEA Therapy Area Market Access Lead for Immunology, Janssen-Cilag Limited, commented “We are pleased with NICE’s decision to accept ustekinumab for use within NHS England and Wales for eligible patients living with ulcerative colitis when either a TNF-alpha inhibitor is not appropriate or has not been effective. We have a long-standing commitment to developing innovative therapies to improve the lives of those living with immune-mediated inflammatory diseases. The decision from NICE reflects the true patient and clinical need for new treatment options for patients living with moderately to severely active ulcerative colitis.”
Ustekinumab has demonstrated a safety profile in UC where trials show the treatment is generally well tolerated. Through to week 44, the proportions of patients receiving ustekinumab (q12w and q8w) were comparable to those receiving placebo in terms of the reporting of at least one adverse event (AE) (69.2%, 77.3% and 78.9%, respectively), serious AEs (7.6%, 8.5% and 9.7% respectively), infections (33.7%, 48.9% and 46.3%, respectively) and serious infections (3.5%, 1.7% and 2.3%, respectively). Overall, the safety for ustekinumab in UC patients was consistent with the known safety profile of ustekinumab in Crohn’s disease (CD).5
Ustekinumab is already recommended by NICE as a clinical and cost-effective option for the treatment of plaque psoriasis, psoriatic arthritis and CD.[vi],[vii],[viii] The FAD will form the basis of the final Technology Appraisal Guidance (TAG) issued to the NHS in England and Wales which is expected to be published in June 2020. Pending any appeals from key stakeholders, based on this positive recommendation, the NHS should make ustekinumab available in England and Wales within three months of publication of the TAG.
*Janssen has provided Crohn’s & Colitis UK with core funding for disease awareness and membership engagement activities. Crohn’s & Colitis UK has not been compensated for any media work.
**Professor Lindsay has received consultancy honoraria from Janssen. He has not been compensated for any media work.
About Ulcerative Colitis (UC)
UC is a chronic disease of the large intestine, also known as the colon, and the rectum, in which the lining becomes inflamed and develops tiny open sores, or ulcers, that produce pus and mucous. UC is the result of an abnormal response by the body's immune system. The symptoms of UC can vary greatly from person to person and can be painful, embarrassing and debilitating, placing a significant burden on those with the condition.[ix],[x] Symptoms may include loose and more urgent bowel movements, persistent diarrhoea, abdominal pain, bloody stools, loss of appetite, weight loss and fatigue.10
About STELARA® (ustekinumab)
In the UK and EU, ustekinumab is approved for the treatment of adult patients with moderately to severely active CD who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a TNF‑alpha antagonist, or have medical contraindications to such therapies. Ustekinumab is also approved for the treatment of adults with moderately to severely active UC who have had an inadequate response with, or lost response to, or were intolerant to either conventional therapy or a biologic, or have medical contraindications to such therapies.[xi]
Ustekinumab is approved alone or in combination with methotrexate (MTX) for the treatment of active psoriatic arthritis in adult patients when the response to previous non-biological disease-modifying antirheumatic drug therapy has been inadequate. Ustekinumab is also approved for the treatment of moderate to severe plaque psoriasis in children and adolescent patients aged six years and older who are inadequately controlled by, or are intolerant to other systemic therapies or phototherapies, and is also approved for the treatment of moderate to severe plaque psoriasis in adults who failed to respond to, have a contraindication to, or are intolerant to other systemic therapies including cyclosporine, MTX or psoralen plus ultraviolet A.11
The Janssen Pharmaceutical Companies of Johnson & Johnson maintain exclusive worldwide marketing rights to STELARA®.
Important safety information
The most common adverse reactions (> 5%) in controlled periods of the adult psoriasis, psoriatic arthritis, Crohn's disease and ulcerative colitis clinical studies with ustekinumab were nasopharyngitis and headache. Most were considered to be mild and did not necessitate discontinuation of study treatment. The most serious adverse reaction that has been reported for ustekinumab is serious hypersensitivity reactions including anaphylaxis. The overall safety profile was similar for patients with psoriasis, psoriatic arthritis, Crohn's disease and ulcerative colitis.11
Please refer to the full Summary of Product Characteristics for full prescribing information for ustekinumab: https://www.medicines.org.uk/emc/product/7638/smpc.
About the UNIFI Programme
UNIFI is a Phase 3 clinical trial, designed to evaluate the safety and efficacy of ustekinumab induction and maintenance dosing for the treatment of moderate to severe UC in adults who demonstrated an inadequate response to, or were unable to tolerate, conventional (i.e., corticosteroids, immunomodulators) or biologic (i.e., one or more TNF blockers and/or vedolizumab) therapies.5
Both the induction and maintenance studies are randomised, double‑blind, placebo-controlled, parallel group, multicentre studies. The induction study was conducted over a duration of at least 8 weeks for each participant. Participants achieving clinical response in the induction study were eligible for the maintenance study.5
The maintenance study was 44 weeks in duration. The primary endpoint of the induction study was clinical remission at week 8, and the primary endpoint for the maintenance study was clinical remission at week 44 among responders to a single IV ustekinumab infusion. Following completion of the maintenance study, a long-term extension study is following eligible participants for an additional three years.5
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.
Cautions Concerning Forward-Looking Statements
This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding NICE’s technology appraisal for STELARA® (ustekinumab). The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen-Cilag Limited, and any of the other Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 29, 2019, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in the company’s most recently filed Quarterly Report on Form 10-Q, and the company’s subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.
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[i] National Institute for Health and Care Excellence. Ustekinumab for treating moderately to severely active ulcerative colitis [ID1511]. Available at: https://www.nice.org.uk/guidance/indevelopment/gid-ta10434/documents. Accessed April 2020.
[ii] Crohn’s & Colitis UK. Who gets Ulcerative Colitis? Available at: https://www.crohnsandcolitis.org.uk/about-inflammatory-bowel-disease/ulcerative-colitis. Accessed April 2020.
[iii] Crohn’s and Colitis Foundation: Treatment Options. Available at: https://www.crohnscolitisfoundation.org/what-is-ulcerative-colitis/treatment-options. Accessed April 2020.
[iv] Toussirot E. The IL23/Th17 pathway as a therapeutic target in chronic inflammatory diseases. Inflamm Allergy Drug Targets 2012;11(2):159–68.
[v] Sands B, et al. Ustekinumab as Induction and Maintenance Therapy for Ulcerative Colitis. N Engl J Med 2019; 381:1201-1214.
[vi] National Institute for Health and Care Excellence. Ustekinumab for the treatment of adults with moderate to severe psoriasis [TA180]. Available at: https://www.nice.org.uk/guidance/indevelopment/ta180/. Accessed April 2020.
[vii] National Institute for Health and Care Excellence. Ustekinumab for treating active psoriatic arthritis [TA340]. Available at: https://www.nice.org.uk/guidance/indevelopment/ta340/. Accessed April 2020.
[viii] National Institute for Health and Care Excellence. Ustekinumab for moderately to severely active Crohn’s disease after previous treatment [TA456]. Available at: https://www.nice.org.uk/guidance/indevelopment/ta456/. Accessed April 2020.
[ix] Crohn’s & Colitis Foundation. ‘Living with Ulcerative Colitis’ leaflet. Available at: https://www.crohnscolitisfoundation.org/sites/default/files/legacy/assets/pdfs/living-with-ulcerative.pdf. Accessed April 2020.
[x] Crohn's & Colitis UK. What is Ulcerative Colitis? Available at: https://www.crohnsandcolitis.org.uk/about-inflammatory-bowel-disease/ulcerative-colitis. Accessed April 2020.
[xi] European Medicines Agency. Stelara SmPC. 2018. https://www.ema.europa.eu/en/documents/product-information/stelara-epar-product-information_en.pdf. Accessed April 2020.