FDA Approves Treatment Option for Polyarticular Course JIA

The US Food and Drug Administration (FDA) has approved Xeljanz (tofacitinib) for the treatment of active polyarticular course juvenile idiopathic arthritis (pcJIA) among pediatric patients aged 2 years or older.

The FDA approved 2 formulations—a tablet and an oral solution—that are dosed according to patients’ weight.

“Although there are already several advanced treatments available, tofacitinib will be an appealing new option given it does not require injections or infusions. These can be quite burdensome to both children with pcJIA and their caretakers,” Hermine Brunner, MD, MSc, director of the Division of Rheumatology at Cincinnati Children’s Hospital Medical Center and scientific director of the Pediatric Rheumatology Collaborative Study Group, said in a press release. “The FDA approval of Xeljanz for pcJIA is positive news for this community as it provides a new advanced treatment option in an oral formulation.”

The approval comes after a Phase 3 study showed that Xeljanz—taken as either a 5 mg-tablet or as a 1 mg/mL-oral solution twice daily based on a patient’s body weight (<40 kg for the oral solution) and/or patient preference—was safe and effective.

The efficacy was displayed in patients with pcJIA who achieved a JIA American College of Rheumatology 30 response at the end of the 18-week open-label, run-in phase. At week 44, those who had been treated with Xeljanz had a statistically significant lower occurrence of disease flare than patients treated with placebo.

The study participants experienced adverse drug reactions that are consistent with those experienced by adult with rheumatoid arthritis. The most common serious adverse events are serious infections, including pneumonia, cellulitis, herpes zoster, urinary tract infection, diverticulitis, and appendicitis.

According to the press release, Xeljanz is the first Janus kinase inhibitor approved in the United States for the treatment of pcJIA.

—Colleen Murphy

Reference:

U.S. FDA approves Pfizer’s XELJANZ® (tofacitinib) for the treatment of active polyarticular course juvenile idiopathic arthritis. News release. Pfizer. September 28, 2020. Accessed September 30, 2020. https://www.pfizer.com/news/press-release/press-release-detail/us-fda-approves-pfizers-xeljanzr-tofacitinib-treatment