FDA Green Lights Tocilizumab for Treatment of Active Polyarticular Juvenile Idiopathic Arthritis

The US Food and Drug Administration (FDA) announced the approval Genentech’s subcutaneous formulation of tocilizumab (Actemra) for treatment of active polyarticular juvenile idiopathic arthritis (PJIA) in patients 2 years of age and older.

The approval is backed by data from the JIGSAW-117 study, a 52-week phase 1b pharmacokinetic/pharmacodynamic bridging study that was designed to determine the appropriate dosing regimen of Actemra subcutaneous across an array of body weights in children with PJIA.

“Polyarticular juvenile idiopathic arthritis is a rare, often painful disease in children,” Sandra Horning, MD, chief medical officer, head of global product development, Genentech, said in a statement. “With this approval, we are please Actemra offers an alternative delivery option to physicians and parents of children aged 2 or older to treat this debilitating disease.”

The study enrolled 52 patients aged 1—17 years old with PJIA who experienced previous inadequate response or intolerance to methotrexate and were either Actemra naïve or received Actemra IV with adequate disease control.

The treatment was administered open label based on a body weight dosing regimen: PJIA patients weighing <30 kg received 162 mg of Actemra every 3 weeks and PJIA patients weighing ≥30 kg received 162 mg every 2 weeks for 52 weeks.

The safety profile was consistent with Actemra IV, with the exception of injection site reactions and neutropenia. A higher frequency of injection site reactions was observed in Actemra subcutaneous treated PJIA patients versus patients treated with Actemra subcutaneous for other approved indications, with a frequency of 28.8% (15/52) injection site reactions observed in Actemra subcutaneous PJIA treated patients.

Injection site reactions were mild and none required patient withdrawal from treatment or dose interruption.

While monitoring patients, a decrease in neutrophil counts below 1 × 10⁹ per L occurred in 15.4% of patients and was more often observed in patients less than 30 kg (25.9%) versus patients at or above 30 kg (4%).

The efficacy in children 2—17 years of age is based on pharmacokinetic exposure and extrapolation of established efficacy of Actemra IV in PJIA patients and Actemra subcutaneous in patients with rheumatoid arthritis.

In 2013, the FDA approved intravenous formulation of Actemra for patients 2 years of age and older with active PJIA.