This week, the FDA approved an expanded indication of the Janus kinase (JAK) inhibitor tofacitinib (Xeljanz) to include adults with moderate to severe ulcerative colitis. Tofacitinib is the first oral medicine approved for long-term use in UC, and provides an alternative to anti–tumor necrosis factor (anti-TNF) drugs that must be injected or infused.
This week, the FDA approved an expanded indication of the Janus kinase (JAK) inhibitor tofacitinib (Xeljanz) to include adults with moderate to severe ulcerative colitis (UC). Tofacitinib is the first oral medicine approved for long-term use in UC, and provides an alternative to anti—tumor necrosis factor (anti-TNF) drugs that must be injected or infused.
The “approval provides an alternative therapy for a debilitating disease with limited treatment options,” said Julie Beitz, MD, director of the Office of Drug Evaluation in FDA’s Center for Drug Evaluation and Research, in a statement.
The approval was based on data collected from 3 controlled clinical trials. In 2 different 8-week placebo-controlled trials, 10 mg of tofacitinib, administered twice daily, resulted in remission in 17% to 18% of patients by week 8. Of these patients, 35% and 47% achieved sustained, corticosteroid-free remission when treated with 5 mg or 10 mg, respectively.
In the final placebo-controlled clinical trial, tofacitinib was administered at a 5-mg or 10-mg dose twice daily. Among patients in the study who achieved a clinical response by week 8, the drug induced remission by week 52 in 34% and 41% of patients, respectively.
The most commonly reported adverse events (AEs) were diarrhea, elevated cholesterol levels, headache, rash, upper respiratory tract infection, herpes zoster, increased blood creatine phosphokinase, and the common cold. Less common serious AEs seen in the studies included malignancy and serious infections, such as opportunistic infections.
However JAK inhibitors as a class have recently come under increased examination after the FDA’s Adverse Events Reporting System revealed 18 primary cases of pulmonary embolism in patients taking tofacitinib.
This heightened incidence of thromboembolic adverse events has also been seen in ruxolitinib (Jakafi), drawing concerns of whether the safety issue may be class-wide for JAK inhibitors. Then, in April 2018, the FDA’s Arthritis Advisory Committee voted in favor of recommending approval of a low dose of the JAK inhibitor baricitinib to treat rheumatoid arthritis, and against recommending approval of a higher dose, because of concerns about safety that included the risk of thrombosis. On June 1, the FDA approved baricitinib as Olumiant at the lower, 2-mg dose, and required a black-box safety warning for thrombosis as well as serious infections and malignancy.
How State Substitution Laws Shape Insulin Biosimilar Adoption
April 15th 2025States with fewer restrictions on biosimilar substitution tend to see higher uptake of interchangeable insulin glargine, showing how even small policy details can significantly influence biosimilar adoption and expand access to more affordable insulin.
How AI Can Help Address Cost-Related Nonadherence to Biologic, Biosimilar Treatment
March 9th 2025Despite saving billions, biosimilars still account for only a small share of the biologics market—what's standing in the way of broader adoption and how can artificial intelligence (AI) help change that?
Early Success of Adalimumab Biosimilars Featured at AMCP 2025
April 5th 2025High adherence rates, comparable clinical effectiveness, and cost savings have marked the early adoption of adalimumab biosimilars in the US, particularly in formulary-driven transitions, as shown in 2 retrospective studies presented at the Academy of Managed Care Pharmacy annual meeting (AMCP 2025).
Will the FTC Be More PBM-Friendly Under a Second Trump Administration?
February 23rd 2025On this episode of Not So Different, we explore the Federal Trade Commission’s (FTC) second interim report on pharmacy benefit managers (PBMs) with Joe Wisniewski from Turquoise Health, discussing key issues like preferential reimbursement, drug pricing transparency, biosimilars, shifting regulations, and how a second Trump administration could reshape PBM practices.
Patients With IBD Maintain Therapy 2 Years Post Switching to Infliximab Biosimilar
March 23rd 2025People with inflammatory bowel disease (IBD) who switched to the infliximab biosimilar CT-P13 had higher treatment persistence (84% and 91%) than those new to infliximab (66% and 53%), with no new safety concerns.