The FDA approved Actemra as a treatment for children two years of age and older with active Systemic Juvenile Idiopathic Arthritis.
that the US Food and Drug Administration (FDA) has approved Actemra (tocilizumab) as a treatment for children two years of age and older with active Systemic Juvenile Idiopathic Arthritis (SJIA) after the drug met its primary phase III endpoints.
SJIA, the rarest type of Juvenile Idiopathic Arthritis (JIA), affects roughly ten to twenty percent of children with JIA, the peak age of onset falling between eighteen months and two years, although it is possible for the disease to persevere into adulthood.
The overall mortality rate for children with SJIA is two to four percent and is responsible for nearly two-thirds of all childhood arthritis deaths.
Actemra is the first drug approved by the FDA for the treatment of SJIA, which possesses the direst long-term prognosis of all types of childhood arthritis.
“As the first and only approved treatment for SJIA, Actemra offers a new option for this extremely difficult to treat disease,” said Hal Barron, MD, chief medical officer and head Global Product Development. “This approval also demonstrates our commitment to science and patients with high unmet medical need, including orphan diseases.”
This study is the first part of a five-year ongoing study.
The study was international and included forty-three sites in seventeen countries, evaluating the efficiency and safety profile of the drug versus the placebo for twelve weeks in 112 children with SJIA.
The average disease duration in the study was five years; participants were children ages two to seventeen years of age with active SJIA persisting for at least six months who could not tolerate or did not respond adequately to previous treatments. They were randomly selected to receive Actemra or a placebo every two weeks as a sixty-minute single intravenous drip infusion for twelve weeks. If the patients were receiving NSAIDs, corticosteroids and methotrexate prior to the commencement of the study, they continued to receive said medicines for the duration of it.
Actemra can be administered solitarily or in combination with methotrexate in patients with SJIA.
It produced significant results: seventy-one percent of the children in the Actemra group achieved a JLA ACR70 response by week twelve compared to the eight percent of children who received the placebo. Also, eighty-five percent participants receiving Actemra experienced a thirty percent improvement in the signs and symptoms of SJIA, reaching a JIA ACR30; they also experienced an absence of fever after twelve weeks, compared with twenty-four percent of children receiving a placebo.
While no new safety signals were identified with Actemra, sixteen percent of patients in the Actemra group and five percent of patients in the placebo group experienced an infusion reaction; anaphylaxis was also reported in one of the patients treated with the drug at some stage in the controlled and open-label extension study.
A little over five percent of the study experienced adverse reactions, the most frequent ones reported in the study being upper respiratory infection, headache, nasopharyngitis and diarrhea. The most commonly reported infections included pneumonia, gastroenteritis, chickenpox, and ear infections.
The primary endpoint of the study was the number of patients treated with Actemra with a JIA ACR30 response and a lack of fever at the final week—week twelve—in comparison to the group receiving the placebo.
“The goal of treatment for children with SJIA is to reduce the signs and symptoms of the disease, including swelling, pain and other complications,” said Hermine Brunner, MD, MSc, study investigator and associate professor of pediatric rheumatology, University of Cincinnati College of Medicine. “We're excited about the results of this study which show that Actemra significantly improved disease signs and symptoms as measured by a JIA ACR response, plus absence of fever, a critical validated efficacy measure of SJIA treatment.”
“Today's FDA approval marks an important advance in the treatment of SJIA,” stated Barron.