Pfizer (PFE) has announced that the US Food and Drug Administration (FDA) has approved Xeljanz (tofacitinib) for the treatment of children and adolescents 2 years plus with active polyarticular course juvenile idiopathic arthritis (pcJIA). Two formulations were approved, a tablet and an oral solution, and are dosed based upon weight.
This approval makes Xeljanz the first and only Janus kinase (JAK) inhibitor approved in the US for the treatment of pcJIA.
Polyarticular JIA is characterized by arthritis in five or more joints and affects both the small joints of the hands and feet, and large joints like the knees, hips and ankles. While its cause is unknown, approximately 300,000 children in the U.S. have a form of JIA.
“Although there are already several advanced treatments available, tofacitinib will be an appealing new option given it does not require injections or 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” said Dr. Hermine Brunner, of Cincinnati Children’s Hospital Medical.
This approval was based on data from a Phase 3 study including two phases: an 18-week open-label, run-in phase (of 225 patients), followed by a 26-week double-blind, placebo-controlled, randomized, withdrawal phase (of 173 patients) for a total duration of 44 weeks.
The trial met its primary endpoint showing that in patients with pcJIA, the occurrence of disease flare in patients treated with tofacitinib (31 percent; n/N=27/88) was statistically significantly (p=0.0007) lower than patients treated with placebo (55 percent; n/N=47/85) at week 44.
In general, the types of adverse drug reactions in patients with pcJIA were consistent with those seen in adult rheumatoid arthritis (RA) patients.
According to Pfizer, Xeljanz oral solution should be available by the end of Q1 2021, while 5mg tablets are available immediately.
Xeljanz is already approved in the U.S. in four indications including for adults with moderately to severely active rheumatoid arthritis and active psoriatic arthritis.
Shares in Pfizer traded marginally higher in Monday’s after-hours trading. On a year-to-date basis the stock is down 7%, but it scores a cautiously optimistic Moderate Buy outlook from the Street. That’s with 4 recent buy ratings offset by 6 hold ratings. Meanwhile the average analyst price target of $43 indicates 19% upside potential lies ahead.
“While peak potential on lead late stage programs may still be debated, core business updates were encouraging and breadth of overall pipeline was clear” commented RBC Capital analyst Randall Stanicky after PFE’s recent investor event.
He reiterated his buy rating on the stock with a $43 price target, telling investors: “We come away with forecasts unchanged but appreciative of where our pipeline assumptions could be conservative.” (See PFE stock analysis on TipRanks).
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