U.S. FDA Approves Pfizer’s Xeljanz (tofacitinib) for the Treatment of Active Polyarticular Course Juvenile Idiopathic Arthritis

NEW YORK–(BUSINESS WIRE) September 28, 2020 — Pfizer Inc. (NYSE: PFE) announced today that the United States (U.S.) Food and Drug Administration (FDA) approved Xeljanz® (tofacitinib) for the treatment of children and adolescents 2 years and older with active polyarticular course juvenile idiopathic arthritis (pcJIA). Two formulations were approved, a tablet and an oral solution, and are dosed based upon weight.¹ This approval makes Xeljanz the first and only Janus kinase (JAK) inhibitor approved in the U.S. for the treatment of pcJIA.

“Polyarticular course juvenile idiopathic arthritis, or pcJIA, is debilitating as it can cause significant jointpain and limit participation in child appropriate activities,” said Dr. Hermine Brunner, Director of the Division of Rheumatology at Cincinnati Children’s Hospital Medical Center and Scientific Director of the Pediatric Rheumatology Collaborative Study Group. “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.”^{2a,2b,3a,4a,5a,5b,5c}

This approval was based on data from a Phase 3 study including two phases: an 18-week open-label, run-in phase (including 225 patients), followed by a 26-week double-blind, placebo-controlled, randomized, withdrawal phase (including 173 patients) for a total duration of 44 weeks. The study evaluated the efficacy and safety of tofacitinib taken as either a 5 mg tablet or as a 1 mg/mL oral solution twice daily based on the subject’s body weight (<40 kg for the oral solution) and/or patient preference.6a,6b The trial met its primary endpoint showing that in patients with pcJIA who achieved a juvenile idiopathic arthritis (JIA) American College of Rheumatology (ACR) 30 response at the end of the run-in phase, 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.^6c,6d In this study, disease flare was defined as a 30 percent or more worsening in at least three of the six variables of the JIA ACR core set, with no more than one of the remaining JIA core response variables improving by 30 percent or more (outcome measures used in JIA clinical trials) after randomization, ^6e,7

In general, the types of adverse drug reactions in patients with pcJIA were consistent with those seen in adult rheumatoid arthritis (RA) patients.Please see important safety information below.

“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,”^4a said Michael Corbo, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development. “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.”

Xeljanz oral solution is anticipated to be available by the end of Q1 2021. Xeljanz 5 mg tablets are available immediately.

About Juvenile Idiopathic Arthritis

Juvenile idiopathic arthritis (JIA) is a chronic inflammatory disease of unknown etiology. JIA includes six categories: systemic, oligoarticular, polyarticular, enthesitis-related, psoriatic, and undifferentiated. 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.2a,2b,3a,8a JIA is defined as arthritis that begins before 16 years of age and persists for at least six weeks.8b While its cause is unknown, approximately 300,000 children in the U.S. have a form of JIA.9,4b

About Xeljanz (tofacitinib)

Xeljanz (tofacitinib) isapproved in the U.S. in four indications: adults with moderately to severely active rheumatoid arthritis (RA) after methotrexate failure, adults with active psoriatic arthritis (PsA) after disease modifying antirheumatic drug (DMARD) failure, adults with moderately to severely active ulcerative colitis (UC) after tumor necrosis factor inhibitor (TNFi) failure, and patients 2 years of age or older with active polyarticular course juvenile idiopathic arthritis (pcJIA). Xeljanz has been studied in more than 50 clinical trials worldwide, including more than 20 trials in RA patients, and prescribed to over 208,000 adult patients (the majority of whom were RA patients) worldwide in the last seven years. ^10,11,12

As the developer of tofacitinib, Pfizer is committed to advancing the science of JAK inhibition and enhancing understanding of tofacitinib through robust clinical development programs in the treatment of immune-mediated inflammatory conditions.

INDICATIONS

Rheumatoid Arthritis

Xeljanz/Xeljanz XR (tofacitinib) is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate.
Limitations of Use: Use of Xeljanz/Xeljanz XR in combination with biologic DMARDs or with potent immunosuppressants such as azathioprine and cyclosporine is not recommended.

Psoriatic Arthritis

Xeljanz/Xeljanz XR (tofacitinib) is indicated for the treatment of adult patients with active psoriatic arthritis who have had an inadequate response or intolerance to methotrexate or other disease-modifying antirheumatic drugs (DMARDs).
Limitations of Use: Use of Xeljanz/Xeljanz XR in combination with biologic DMARDs or with potent immunosuppressants such as azathioprine and cyclosporine is not recommended.

It is important to note that a dosage of Xeljanz 10 mg twice daily is not recommended for the treatment of rheumatoid arthritis or psoriatic arthritis.

Ulcerative Colitis

Xeljanz is indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC), who have had an inadequate response or who are intolerant to TNF blockers.
Limitations of Use: Use of Xeljanz in combination with biological therapies for UC or with potent immunosuppressants such as azathioprine and cyclosporine is not recommended.

Polyarticular Course Juvenile Idiopathic Arthritis

Xeljanz/Xeljanz Oral Solution is indicated for the treatment of active polyarticular course juvenile idiopathic arthritis (pcJIA) in patients 2 years of age and older.
Limitations of Use: Use of Xeljanz/Xeljanz Oral Solution in combination with biologic DMARDs or potent immunosuppressants such as azathioprine and cyclosporine is not recommended.

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DISCLOSURE NOTICE: The information contained in this release is as of September 28, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about Xeljanz (tofacitinib) and a new indication for the treatment of children and adolescents 2 years and older with active polyarticular course juvenile idiopathic arthritis (pcJIA), including their potential benefits and the anticipated timing of availability of XELJANZ oral solution, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; uncertainties regarding the commercial success of Xeljanz and Xeljanz XR; whether and when applications for Xeljanz for pcJIA will be filed in any other jurisdictions and whether and when applications for Xeljanz for any other indications may be filed in any jurisdictions; whether and when any applications that may be pending or filed for any potential indications for Xeljanz or Xeljanz XR in any jurisdictions may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy, and, if approved, whether they will be commercially successful; uncertainties regarding the commercial impact of actions by regulatory authorities based on analysis of clinical trial A3921133 or other data, which will depend, in part, on labeling determinations; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Xeljanz and Xeljanz XR; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

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1 XELJANZ Label. September 2020.
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