Over the past year, the U.S. Food and Drug Administration (FDA) has approved several new rheumatologic drugs for children and adults. These approvals, along with safety signals reported during the last 12 months, were discussed at the annual 2026 Rheumatology Update from the FDA. A recording of the session will be available to registered participants of ACR Convergence 2026 for on-demand viewing.
Jeong Min Yu, MD, MS, Medical Officer at the FDA, opened the session with a review of the new FDA approvals and labeling changes relevant to rheumatology. New adult approvals for the year included brepocitinib, a Janus kinase (JAK) and tyrosine kinase 2 (TYK2) inhibitor, for dermatomyositis, and tildrakizumab, an interleukin-23 (IL-23) antagonist, for psoriatic arthritis (PsA).
Approval of brepocitinib was based on a randomized, double-blind, placebo-controlled trial of 241 adult patients with dermatomyositis. At week 52, the mean total improvement score was 47.5% with the approved 30 mg dose, compared with 33.3% with placebo. Brepocitinib carries the same class-wide boxed warnings as other JAK inhibitors. Other labeled precautions include hypersensitivity, gastrointestinal perforation, hypoglycemia in patients with diabetes, laboratory abnormalities, immunizations, and embryofetal toxicity.
Tildrakizumab was initially approved for moderate-to-severe plaque psoriasis (PsO). The new approval for adult patients with active PsA was based on two randomized, double-blind, placebo-controlled studies in 806 adult patients with active PsA. In both trials, subjects treated with tildrakizumab 100 mg every 12 weeks showed a greater clinical response, as assessed by 20% improvement in American College of Rheumatology criteria (ACR20) at week 24, than subjects treated with placebo. The warnings and precautions include hypersensitivity, infections, pretreatment evaluation for tuberculosis, and immunizations.
New pediatric approvals include secukinumab in patients aged 12 years and older and risankizumab for PsA in patients aged 6 years and older. Secukinumab, an IL-17A antagonist, was initially approved for ankylosing spondylitis in adults. Pediatric approval was based on extrapolation of efficacy from adult patients. Risankizumab, an IL-23 antagonist, was initially approved for PsA in adults. The pediatric PsA indication is supported by adult PsA efficacy data with pharmacokinetic safety information from pediatric PsO studies.
“The foundation for a pediatric extrapolation approach is reliant on the degree of similarities between the adult and pediatric condition,” said Tresa Ambooken, MD, MBA, Medical Officer at the FDA. “Factors taken into account to determine similarity include comparisons of disease etiology and clinical progression, diagnostic criteria, clinical outcome assessments, short- and long-term study endpoints, drug pharmacology, and response to treatment.”
Regarding new routes of administration, subcutaneous anifrolumab was approved for adults with moderate-to-severe systemic lupus erythematosus (SLE) who are receiving standard therapy. The approved subcutaneous regimen is 120 mg once weekly. The efficacy of subcutaneous anifrolumab was evaluated in a randomized, double-blind, placebo-controlled study of 367 adults with moderate-to-severe SLE on background standard-of-care therapies. At week 52, BILAG-based Composite Lupus Assessment (BICLA) response was 58.5% with anifrolumab versus 43.2% with placebo, an adjusted difference of 15.3 percentage points. Overall, the safety profile of subcutaneous anifrolumab was similar to that of intravenous anifrolumab.
Regarding expanded efficacy claims, guselkumab was approved for inhibition of structural joint damage progression. Guselkumab, an IL-23 antagonist, was initially approved for active PsA. The expanded efficacy claim was approved based on two randomized, double-blind, placebo-controlled studies of PsA patients with inadequate response to standard of care. The change in modified van der Heijde-Sharp score at week 24 reached statistical significance in one trial. The warnings and precautions for guselkumab did not change significantly with its expanded efficacy claims.
Over-the-counter approvals include acetaminophen and naproxen. Biosimilar and interchangeable approvals include Immgolis, Zimrixby, Truxima, and Stoboclo. Generic approvals include tofacitinib.
Karmela Kim Chan, MD, Medical Officer at the FDA, concluded the session with a review of important safety updates from the past year. The FDA officially discontinued the Mycophenolate Risk Evaluation and Mitigation Strategy (MREMS) program for mycophenolate.
“We continue to monitor the safety profile of drugs throughout the life cycle, and part of that work relies on post-marketing reporting, including from all of you in the audience,” Dr. Chan said. “Let me stress that your reporting helps us become aware of safety concerns so that we can take appropriate regulatory action.”
THERE’S STILL TIME TO JOIN US
If you haven’t registered for ACR Convergence 2026, you can still register on-site at the Orange County Convention Center in Orlando, Florida, through November 11. All registered participants receive on-demand access to scientific sessions through September 30, 2027.
