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Rheumatol Ther. 2019 Dec;6(4):503-520. doi: 10.1007/s40744-019-00167-6. Epub 2019 Aug 13.

Two-Year Safety and Effectiveness of Peficitinib in Moderate-To-Severe Rheumatoid Arthritis: A Phase IIb, Open-Label Extension Study.

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Stanford University, Palo Alto, CA, USA.
Desert Medical Advances, Palm Desert, CA, USA.
Hospital Civil de Guadalajara FAA, CUCS UdG, Guadalajara, Mexico.
Centro de Investigación Clínica de Morelia SC, Morelia, Mexico.
Arthritis Associates, Orlando, FL, USA.
ARS Rheumatica sp.z.o.o, Reumatika, Warsaw, Poland.
Health Research of Oklahoma, Oklahoma City, OK, USA.
Astellas Pharma Global Development, Inc., Northbrook, IL, USA.
Altoona Center for Clinical Research, Duncansville, PA, USA.



Peficitinib is a novel orally bioavailable, once-daily Janus kinase (JAK) inhibitor approved in Japan for the treatment of rheumatoid arthritis (RA). This 2-year extension study of two global phase IIb trials investigated the long-term safety and effectiveness of peficitinib.


All eligible patients with moderate-to-severe RA including patients in the placebo group who participated in one of two global phase IIb trials ('with methotrexate' or 'without methotrexate') were included in this 2-year open-label extension study and were converted to peficitinib 100 mg once daily. The primary objective was to evaluate an additional 2 years of safety by assessing treatment-emergent adverse events (AEs) and clinical laboratory evaluations for 105 weeks. Evaluation of an additional 2 years of effectiveness using American College of Rheumatology (ACR) 20/50/70 responses was the exploratory objective.


Overall, 611 patients were enrolled in the extension study: 319 (52.2%) patients completed the study and 292 (48%) discontinued treatment, including for withdrawal of patient consent (n = 96), failure to achieve low disease activity (n = 62), and AE not including death (n = 41). AEs were reported in 463 (76%) patients. The most common AEs (per 100 patient-years) were upper respiratory tract infections (9.9) and urinary tract infections (7.2). Serious AEs were reported in 80 (13%) patients, with incidences per 100 patient-years of serious infections 2.7, herpes zoster 1.5 (including one herpes zoster ophthalmic), and malignancies 0.6 (most frequently basal cell carcinoma). At week 105, 269 (44%) patients demonstrated an ACR20 response relative to their respective phase IIb trial baselines.


Among 319 patients who completed this 2-year extension of two global phase IIb studies, peficitinib 100 mg once daily demonstrated a stable safety profile and sustained effectiveness in patients with moderate-to-severe RA.

TRIAL REGISTRATION: identifier, NCT01711814. Registered 19 October 2012.


Astellas Pharma Global Development, Inc.


Janus kinase (JAK) inhibitor; Long-term extension; Peficitinib; Rheumatoid arthritis

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