FDA Approves Subcutaneous Actemra for Systemic Juvenile Idiopathic Arthritis

Officials with the FDA have approved the subcutaneous (SC) formulation of tocilizumab (Actemra, Genentech) for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients 2 years of age and older, the company announced in a press release.

The intravenous (IV) formulation of the treatment was approved by the FDA with the same indication in 2011. Tocilizumab is also indicated for the treatment of active polyarticular juvenile idiopathic arthritis in patients 2 years of age and older, as well as for the treatment of adults with moderately to severely active rheumatoid arthritis (RA) and giant cell arteritis.

Tocilizumab’s newest formulation offers an additional administration option for children diagnosed with SJIA, a rare and debilitating disease. SJIA, the rarest form of juvenile idiopathic arthritis (JIA), accounts for approximately 10% of the 300,000 JIA cases in the United States. Symptoms of the disease typically include inflammation in 1 or more joints and a daily fever for at least 2 weeks, which may be accompanied by a skin rash.

This approval is based on results from the JIGSAW-118 phase 1b study designed to determine the appropriate dosing regimen if the SC formulation of tocilizumab across a range of body weights in children with SJIA. The study included 51 patients aged 1 to 17 years old with the disease and inadequate response to NSAIDs and corticosteroids who were either tocilizumab naïve or were receiving tocilizumab IV with adequate disease control.

For the trial, patients with SJIA who weighed <30 kg received 162 mg of tocilizumab every 2 weeks or 10 days and patients weighing ≥30 received 162 mg of tocilizumab every week for 52 weeks.

According to the press release, the efficacy of tocilizumab SC in children aged 2 to 17 years old is based on pharmacokinetic exposure and extrapolation of established efficacy of the IV formulation in patients with SJIA and tocilizumab SC in patients with RA.

The safety profile of tocilizumab SC was consistent with the IV formulation, with injection site reactions being the most common adverse effects. Forty-one percent of patients treated with tocilizumab SC experienced injection site reactions compared with patients treated with tocilizumab SC for other approved indications.

“Systemic juvenile idiopathic arthritis is a rare, debilitating disease with limited treatment options,” Sandra Horning, MD, Genentech chief medical officer and head of Global Product Development, said in the press release. “We are pleased to now offer physicians the flexibility to prescribe for children 2 years of age and older either Actemra IV, administered in a medical office, or Actemra subcutaneous, a prefilled syringe that can be injected at home.”

Tocilizumab can be given alone or in combination with methotrexate in patients with SJIA, according to Genentech.

Reference

FDA Approves Subcutaneous Formulation of Actemra for Use in Active Systemic Juvenile Idiopathic Arthritis (SJIA), a Rare Form of Juvenile Arthritis [news release]. Genentech’s website. https://www.gene.com/media/press-releases/14743/2018-09-13/fda-approves-subcutaneous-formulation-of. Accessed September 13, 2018.