The U.S. Food and Drug Administration (FDA) has approved Genentech’s formulation of Actemra (tocilizumab) by subcutaneous injection for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in children ages 2 and older.
The FDA already had approved the use of intravenous Actemra (Actemra IV) in 2011. Actemra IV is administered in a clinic or doctor’s office, which is less convenient for patients and caregivers than Actemra SC, a subcutaneous injection that can be delivered at home.
The approval is based on results of the JIGSAW-118 clinical trial (NCT01904292). The study evaluated Actemra SC’s safety, pharmacokinetics, and pharmacodynamics — how long the medication lasts in the body, its distribution, and activity — when administered over a 52-week period.
The JIGSAW-118 trial was an open-label, multicenter, Phase 1b study to determine the appropriate dose regimen of subcutaneous Actemra across a range of body weights in children with SJIA.
It enrolled 51 patients with SJIA, ages 1 to 17, who had an inadequate response to nonsteroidal anti-inflammatory drugs and corticosteroids. Eligible participants either had never received Actemra before or were receiving intravenous Actemra with adequate disease control.
The safety profile for Actemra SC was similar to that of Actemra IV, according to Genentech. The main difference was the occurrence of injection site reactions with Actemra SC. More Actemra SC-treated patients had these reactions — 21 out of 51, or 41 percent — than patients treated with Actemra SC for other approved indications. The reactions were not serious and did not require withdrawal or interruptions in treatment.
“Systemic juvenile idiopathic arthritis is a rare, debilitating disease with limited treatment options,” Sandra Horning, MD, Genentech’s chief medical officer and head of global product development, said in a press release.
“We are pleased to now offer physicians the flexibility to prescribe for children two 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,” Horning added.
SJIA is the rarest form of juvenile idiopathic arthritis, accounting for about 10 percent of the cases.
In May, the FDA approved Actemra subcutaneous formulation as a treatment for patients 2 and older with active polyarticular juvenile idiopathic arthritis (PJIA). PJIA accounts for about 30 percent of all cases of juvenile idiopathic arthritis and is characterized by inflammation in five or more joints.
The new Actemra SC formulation can be used alone or in combination with standard-of-care methotrexate.