A recent study has filled a research hole, but that may not be enough to get this key drug on the paediatric market.
New research has established the efficacy and safety of a drug for two paediatric arthritis sub-types, but making it available to children with active disease could still be difficult.
Researchers used a phase III clinical trial to demonstrate the efficacy and safety of secukinumab in paediatric patients with active enthesitis-related arthritis and juvenile psoriatic arthritis (ERA and JPsA) but with inadequate response to conventional therapy.
“There remains a high unmet medical need for therapies indicated for ERA and JPsA as there is a dearth of tested therapies,” the authors of the research paper wrote.
The JUNIPERA study, published in the Annals of the Rheumatic Diseases earlier this month, was conducted by Dr Hermine Brunner at the University of Cincinnati, Dr Ivan Foeldvari in Hamburg, Germany, and Dr Ekaterina Alexeeva at the Ministry of Health of the Russian Federation.
“The ERA and JPsA is a really difficult subtype,” said Associate Professor Jane Munro, a paediatric rheumatologist at Royal Children’s Hospital in Melbourne.
“It often takes a long time to present or actually get diagnosed, so out of all the subtypes, it’s the one that has the slowest progression through the healthcare system until it gets to a paediatric rheumatologist.”
But the researchers said they now considered secukinumab, the IL-17A inhibitor, as “an important new treatment option” for children with enthesitis-related arthritis and juvenile psoriatic arthritis, including in the resolution of dactylitis and enthesitis.
The trial was a randomised, placebo-controlled, treatment-withdrawal study.
Biologic-naïve patients, aged between two and 17 years and with active disease, were treated with open-label subcutaneous secukinumab during a 12-week period, designated treatment period 1 (TP1).
Patients who flared in the following treatment period 2 (TP2), immediately entered open-label secukinumab for treatment period 3 (TP3), which lasted up to week 104 of the trial.
The study’s primary endpoint was the time taken for the disease to flare in treatment period 2.
A total of 86 patients, with a median age of 14 years, entered open-label secukinumab in TP1. In TP2, 44 ERA responders and 31 JPsA responders received either secukinumab or a placebo.
The study met its primary endpoint, and the researchers found a statistically significant difference between the secukinumab and the placebo cohorts in the time taken for the disease to flare in TP2.
“Secukinumab was found to result in rapid improvement of arthritis, dactylitis and enthesitis in children with ERA and JPsA,” the paper said.
“The study demonstrated that time to disease flare in TP2 was significantly longer with secukinumab treatment than with placebo in the overall JIA population.”
No new safety signals were reported with secukinumab for up to two years.
“The study design is interesting,” Professor Munro said. “It’s clever and it’s appropriate, the way they’ve had everyone on open label initially, so that everyone had access to it and then they had withdrawal and then [the primary endpoint] was time to flare.
The authors said treatment options for patients with the ERA and JPsA sub-types were limited.
“Conventional synthetic DMARDs, glucocorticoids and nonsteroidal anti-inflammatory drugs provide limited efficacy, with safety issues with long-term use.
“Studies have shown the efficacy of biologic DMARD anti-TNF agents in patients with ERA or JPsA, but many patients continue to experience uncontrolled disease or experience treatment-related side effects.”
But despite the promising findings of the study, the relative rarity of the sub-types could be a barrier to their availability in the Australian market.
“In terms of a practical outcome from this study for Australian children, it’s going to be about how we actually get access to this drug,” Professor Munro said.
“From a compassionate supply perspective, we might be able to, but getting it [PBS] listed is a long way off, I think.”
