Pfizer has secured the US Food and Drug Administration (FDA) approval for XELJANZ (tofacitinib), in tablet and oral solution formulations, to treat a type of polyarticular course juvenile idiopathic arthritis (pcJIA).

The FDA approval allows XELJANZ to be used in the treatment of pcJIA in children aged two years and above as well as adolescents.

XELJANZ is the first and only approved Janus kinase (JAK) inhibitor available in the country for the treatment of pcJIA, claimed the company.

pcJIA is a condition of arthritis in five or more joints and affects both the small joints of the hands and feet, and large joints such as knees, hips and ankles. pcJIA is one of the six categories of chronic inflammatory disease of unknown etiology called juvenile idiopathic arthritis.

Pfizer inflammation and immunology global product development Michael Corbo said: “Many children and adolescents living with polyarticular course juvenile idiopathic arthritis, or pcJIA, are in need of advanced oral treatment options, so we are proud to now offer XELJANZ to this patient community.

“This approval, which is the fourth indication for XELJANZ, reinforces its utility in the treatment of immune-mediated inflammatory conditions and further demonstrates our expertise in JAK science.”

Tablets available now, but oral solution expected to be available by Q1 2021 end

The FDA approval of XELJANZ was supported by the data from Phase 3 study to evaluate the efficacy and safety of the drug, administered either as 5mg tablet or as 1mg/mL oral solution based on the body weight of the subject or patient preference.

The study included two phases – one an open-label, run-in phase involving 225 patients for 18 weeks, followed by a double-blind, placebo-controlled, randomised, withdrawal phase including 173 patients for 26 weeks.

XELJANZ achieved the primary end point indicating that the occurrence of disease flare in patients treated with tofacitinib was statistically significantly lower than patients treated with placebo at week 44.

The kinds of adverse drug reactions seen in patients with pcJIA were consistent with those in adult rheumatoid arthritis (RA) patients.

The US drugmaker is planning to make the oral solution by the end of Q1 2021, while the 5mg tablets are available now.

XELJANZ has been earlier approved in the US to treat adults suffering with rheumatoid arthritis (RA) after methotrexate failure, adults with active psoriatic arthritis (PsA) after disease modifying antirheumatic drug (DMARD) failure, and adults with moderately to severely active ulcerative colitis (UC) after tumor necrosis factor inhibitor (TNFi) failure.

Pediatric Rheumatology Collaborative Study Group scientific director Hermine Brunner said: “Polyarticular course juvenile idiopathic arthritis, or pcJIA, is debilitating as it can cause significant jointpain and limit participation in child appropriate activities.

“Although there arealready several advanced treatments available, tofacitinib will be an appealing new option given it does not require injectionsor infusions.

“These can be quite burdensome to both children with pcJIA and their caretakers. The FDA approval of Xeljanz for pcJIA is positive news for this community as it provides a new advanced treatment option in an oral formulation.”