New phase III results for Pfizer’s Xeljanz in ulcerative colitis (UC) put the company on course to file for approval in a disease that affects millions of people worldwide.
Xeljanz (tofacitinib) is already approved to treat rheumatoid arthritis (RA) but has failed to grow as quickly as hoped in that setting, so the US pharma giant is hoping new indications will help the product meet its blockbuster sales expectations.
Once tipped as a $3bn drug, Xeljanz has been compromised since its launch in 2012 by the FDA’s decision to approve only a 5mg twice-daily dose in RA and ask for more evidence on the safety of a higher 10mg dose.
Safety concerns also prevented approval for RA in Europe, and Pfizer hit another major setback last November after the FDA turned down Xeljanz as a psoriasis therapy – though action for the chronic plaque psoriasis supplemental New Drug Application was laid out in October.
The latest data from the OCTAVE Sustain trial backs the use of the drug as a maintenance therapy for patients with ulcerative colitis – a debilitating inflammatory bowel disease that can cause abdominal pain, fever, weight loss and chronic, bloody diarrhoea.
Previously, Pfizer reported two positive phase III trials of Xeljanz – OCTAVE Induction 1 and 2 – which showed that the drug was able to bring patients with moderate-to-severe active UC into remission more effectively than placebo.
OCTAVE Sustain compared the 5mg and 10mg doses of Xeljanz to placebo in patients already in remission from UC, and showed that both doses were able to reduce the risk of relapse significantly. Detailed results from the study will be presented at a future medical conference, said Pfizer.
The company is also conducting a long-term, open-label extension study of patients enrolled in its three main phase III trials and will also include data from this trial in marketing applications, currently scheduled for the first half of 2017.
Xeljanz has started to gain momentum, with sales up 66% to $172m last year, but will need a major boost from new indications if it is to push above the $1bn sales threshold.
In addition to UC, the FDA approved Xeljanz XR (tofacitinib citrate) extended-release 11 mg tablets for the once-daily treatment of moderate to severe RA in patients who have had an inadequate response or intolerance to methotrexate in February.