The US Food and Drug Administration (FDA) has approved the subcutaneous (SC) formulation of tocilizumab (Actemra) for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients aged 2 years of age and older. Tocilizumab can be administered alone or in combination with methotrexate (MTX).
The intravenous formulation of tocilizumab was previously approved for patients aged 2 years of age and older with active SJIA.
“Systemic juvenile idiopathic arthritis is a rare, debilitating disease with limited treatment options,” said Sandra Horning, MD, chief medical officer and head of Global Product Development, in a recent statement
. “We are pleased to now offer physicians the flexibility to prescribe for children two years of age and older either tocilizumab IV, administered in a medical office, or tocilizumab subcutaneous, a prefilled syringe that can be injected at home.”
The JIGSAW-118 study, a 52-week, open-label, multicenter, Phase 1b pharmacokinetic (PK)/pharmacodynamic (PD) bridging trial served as the basis for the approval. The appropriate dosing regimen of tocilizumab SC was determined by the trial across a range of body weights (BWs) in pediatric patients with SJIA.
Fifteen patients with SJIA aged 1 to 17 years were enrolled in the trial. Enrollment criteria also included those with inadequate response to nonsteroidal anti-inflammatory drugs (NSAIDs) and corticosteroids who were either tocilizumab naive or were receiving tocilizumab IV with adequate disease control. Patients received tocilizumab according to a body weight –based dosing regimen, with SJIA patients weighing <30 kg receiving 162 mg of tocilizumab every 2 weeks or 10 days; and SJIA patients weighing ≥30 kg receiving 162 mg of tocilizumab every week for 52 weeks. Safety, model-computed PK and PD parameters were assessed.
Except for injection site reactions (ISRs), tocilizumab SC’s safety was observed to be consistent with the known safety profile of tocilizumab IV. Forty-one percent (21/51) of tocilizumab SC treated patients (a higher frequency) experienced ISRs. However, no patients withdrew from treatment or experienced dose interruption, and all ISRs reported were non-serious.
"The FDA approval of the subcutaneous formulation of Actemra for patients with sJIA enables the flexibility for children to be treated at home, instead of an infusion center – this minimizes the impact of treatment on the schedules of patients, as well as their families," said Páris Sidiropoulos, PhD, Senior Medical Science Director, Immunology/US Medical Affairs, Genentech, to Rare Disease Report®.
Tocilizumab SC’s efficacy in pediatric patients aged 2 to 17 years of age is grounded in PK exposure and extrapolation of established efficacy of tocilizumab IV in SJIA patients and tocilizumab SC in patients with rheumatoid arthritis (RA).