The U.S. Food and Drug Administration (FDA) has agreed to the expanded use of Enbrel (etanercept) to treat children with chronic moderate-to-severe plaque psoriasis, making Amgen’s biologic the first systematic therapy approved for pediatric patients ages 4 and older.
A supplemental Biologics License Application (sBLA) for Enbrel, submitted on Jan. 5, drew on results of a one-year, placebo-controlled Phase 3 clinical trial (NCT00078819) and its five-year open-label extension study (NCT00141921) evaluating the safety and efficacy of Enbrel in patients, ages 4 to 17, with chronic moderate-to-severe plaque psoriasis.
Results showed significant treatment efficacy — study endpoints included rates of 75% and 90% improvement in Psoriasis Area and Severity Index score and clear/almost clear on static physician global assessment — and adverse events similar to those seen in treated adult populations.
“As many parents of children with moderate-to-severe plaque psoriasis can tell you, there is a need for FDA approved systemic therapies in the pediatric setting. Until now, no biologics — which are effective in treating adults with moderate-to-severe plaque psoriasis — had been approved in the U.S. for the treatment of … children,” Randy Beranek, president and chief executive officer of the National Psoriasis Foundation, said in a press release. “This new approval is an important development for this patient community, as well as their parents and families, and marks a significant milestone in advancing the treatment of children living with this devastating disease.”
In the long-term extension study, 181 patients received Enbrel and 69 remained under treatment for 264 weeks (about five years). Final results showed Enbrel treatment was significantly effective, meeting its goals of 75% improvement (60% to 70% of all patients) and 90% improvement (30% to 40%) on Psoriasis Area and Severity scale scores. Almost 89% of pediatric patients, or 161, also reported adverse events, the most common being upper respiratory tract infection, nasopharyngitis, and headache, but no opportunistic infections or malignancies. These events were similar to those observed in previous clinical trials in adult patients.
“The need for an effective treatment for chronic moderate-to-severe pediatric psoriasis patients is high, and safety is always a concern when it comes to treating children. ENBREL has over a decade of experience in adult moderate to severe plaque psoriasis, and that proven track record matters,” said Sean E. Harper, MD, executive vice president of Research and Development at Amgen. “Today’s FDA approval shows that innovation doesn’t stop with a drug’s first market approval.”
Enbrel, produced by Amgen, treats autoimmune diseases by interfering with tumor necrosis factor (TNF; a soluble inflammatory cytokine), acting as a TNF inhibitor. It is was first approved by the FDA to treat moderate-to-severe rheumatoid arthritis (1998), and subsequently approved as a treatment for moderate-to-severe polyarticular juvenile idiopathic arthritis (1999), psoriatic arthritis (2002), ankylosing spondylitis (2003), and adults with moderate-to-severe plaque psoriasis (2004).
About one-third of all psoriasis cases are reported in children.