Janssen’s Stelara® approved for expanded use by European Commission

Janssen's Stelara® approved for expanded use by European Commission

Janssen’s Stelara ® (ustekinumab) has been approved for expanded use by the European Commission. The expanded use of ustekinumab is for the treatment of adult patients with moderately to severely active ulcerative colitis (UC), who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic or have medical contraindications to such therapies.

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 and an important therapeutic target in UC.

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. The symptoms of UC can vary greatly from person to person and can be painful, embarrassing and debilitating, placing a significant burden on people with the condition. For up to two-thirds of people with UC, current treatments are not completely successful or complications arise.

The EC approval is based on data from the pivotal Phase 3 UNIFI trial programme – a programme that was split into an initial Induction study (UNIFI-I) of eight weeks, followed by a Maintenance study (UNIFI-M) of 44 weeks – both of which demonstrated ustekinumab’s safety and efficacy as a treatment option for patients with moderately to severely active UC who demonstrated an inadequate response to or were unable to tolerate conventional (i.e. corticosteroids or immunomodulators) or biologic (i.e. one or more tumour necrosis factor [TNF]-alpha antagonists and/or vedolizumab) therapies.

Ustekinumab has demonstrated a favourable safety profile in UC where trials show the treatment is generally well tolerated. In the primary randomised population of the induction and maintenance studies, a similar proportion of patients in the ustekinumab and placebo groups experienced adverse events (AE), serious AEs, infections and serious infections through to week 44. During the induction phase, one death from an oesophageal varices haemorrhage was reported, and no malignancies, opportunistic infections or tuberculosis were reported. During the maintenance phase, no deaths and two malignancies other than non‑melanoma skin cancer (NMSC) were reported (90 mg ustekinumab q8w: colon cancer [n=1]; 90 mg ustekinumab q12w: papillary renal cell carcinoma [n=1]). There was one patient-reported NMSC in the 90 mg ustekinumab q12w group (2 squamous cell carcinoma events).

Marketing authorisation follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), issued on 25 July 2019.

In January, Janssen completed an EMA application for Stelara ® for ulcerative colitis.