The CHMP has adopted a positive opinion recommending marketing authorisation in the EU for the use of ustekinumab 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.
“Ulcerative colitis is a particularly disruptive, life-long condition with unpredictable flares that can negatively impact every aspect of patients’ lives. More treatments that offer long-term relief are urgently needed as, despite currently available treatment options, many people with the condition continue to suffer symptoms,” said Jaime Oliver, MD, Janssen Therapeutic Area Lead, Immunology, Europe, Middle East & Africa, Cilag GmbH International. “We welcome the CHMP’s positive opinion on ustekinumab, which brings us one step closer to making this important medicine available for people with ulcerative colitis in Europe, potentially offering them a chance of durable remission and symptomatic relief.”
Ustekinumab is the first treatment to selectively target the IL-12/IL-23 pathway, an important therapeutic target in UC. The CHMP adopted the opinion based on data from the pivotal Phase III UNIFI trial programme which demonstrated ustekinumab’s 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 (for example, corticosteroids or immunomodulators) or biologic (tumour necrosis factor (TNF)-alpha antagonists or vedolizumab) therapies.
Ustekinumab has demonstrated a favourable safety profile in UC where trials show the treatment is 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 UNIFI Induction phase one death from an oesophageal varices haemorrhage was reported, and no malignancies, opportunistic infections or tuberculosis were reported. During the UNIFI Maintenance phase, no deaths and two malignancies other than non‑melanoma skin cancer (NMSC) were reported (90mg ustekinumab q8w: colon cancer [n=1]; 90mg ustekinumab q12w: papillary renal cell carcinoma [n=1]). There was one patient-reported NMSC in the 90mg ustekinumab q12w group (two squamous cell carcinoma events).
Following this positive opinion, a final decision from the European Commission regarding its marketing authorisation is expected later this year.