Roche‘s (OTC:RHHBY) Genentech reported this week that the FDA approved the subcutaneous formulation of tocilizumab for the treatment of active systemic juvenile idiopathic arthritis in patients two years and older.
The U.S. agency approved the intravenous formulation of Actemra for the same indication in 2011.
Systemic juvenile idiopathic arthritis affects roughly 30,000 children in the U.S., according to Roche. The condition brings about inflammation in one or more joints and a daily fever for at least two weeks. The company noted that the symptoms of SJIA can also include anemia, enlargement of the spleen or liver and inflammation of the lining of the heart and lungs.
“Systemic juvenile idiopathic arthritis is a rare, debilitating disease with limited treatment options,” Dr. Sandra Horning, chief medical officer & head of global product development, said in prepared remarks. “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.”
Roche’s approval for the subcutaneous formulation of Actemra was based on data from a 52-week Phase Ib pharmacokinetic/pharmacodynamic bridging study that evaluated the drug’s appropriate dosing regimen in children with SJIA. The 51-patient study included people with SJIA who were not responding to NSAIDs and corticosteroids.
The safety profile for the subcutaneous formulation of Actemra was consistent with the known safety profile of Actemra IV, Roche said, except for injection site reactions.
The efficacy of the subcutaneous formulation was determined based on PK exposure and extrapolation of the established efficacy of the IV formulation in SJIA patients, as well as the subcutaneous formulation of Actemra in patients with rheumatoid arthritis, Roche reported.