The FDA has approved tofacitinib as a treatment for juvenile patients with active polyarticular course juvenile idiopathic arthritis.
According to a press release from the manufacturer, the approval followed a phase 3 study, including an 18-week open-label phase and a 26-week double-blind, randomized phase. During the 44 weeks of research, the trial evaluated the safety and efficacy of tofacitinib. The release noted that the trial reached its primary endpoint of patients who achieved ACR 30 response at the end of the run-in phase, the occurrence of disease flare in the tofacitinib arm (31% n/N=27/88) was significantly (p=0.0007) lower than patients treated with placebo (55%; n/N=47/85) at week 44.
During the trial, disease flare was defined as a 30% or more worsening in at least three of six JIA ACR core set variables, with no more than one of the remaining variables improving by 30% or more after randomization.
“Polyarticular course juvenile idiopathic arthritis, or PcJIA, is debilitating as it can cause significant joint pain and limit participation in child appropriate activities,” said Hermine I. Brunner, MD, MSc, MBA, Director of the Division of Rheumatology at Cincinnati Children’s Hospital Medical Center. “Although there are already several advanced treatments available, tofacitinib will be an appealing new option given that it does not require injections or infusions. This can be quite burdensome to both children with pcJIA and their caretakers.”
The release noted that during clinical trials with rheumatoid arthritis patients, the most common adverse reactions in the first three months were upper respiratory tract infection, nasopharyngitis, diarrhea, headache, and hypertension.
The approval of tofacitinib allows the drug to be administered either as a tablet or an oral solution, based on weight, which Brunner called “positive news for this community.” With the approval, the tablet form of the medication is available immediately. The oral solution is expected to reach the market by the end of the first quarter of 2021.