Rare Disease Report

FDA Approves Subcutaneous Formulation of Tocilizumab for Rare Form of Juvenile Arthritis

SEPTEMBER 13, 2018
Rare Disease Report® Editorial Staff
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).

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