The study’s key secondary end points were JIA ACR50/70 response rates and the change from phase 2 baseline in CHAQ-DI score at week 44.
Patients received tofacitinib citrate twice daily either as a 5 mg tablet or as a 1 mg/mL oral solution. The treatment form was based on the patient’s weight. If patients weighed less than 40 kg, they received the oral solution; if they weighed 40 kg or more, they received the tablet. The weight-based, twice-daily dosing was:
- 5 kg to <7 kg: 2 mg or 2 mL of tofacitinib citrate;
- 7 kg to <10 kg: 2.5 mg or 2.5 mL of tofacitinib citrate;
- 10 kg to <15 kg: 3 mg or 3 mL of tofacitinib citrate;
- 15 kg to <25 kg: 3.5 mg or 3.5 mL of tofacitinib citrate;
- 25 kg to <40 kg: 4 mg or 4 mL of tofacitinib citrate;
- ≥40 kg: 5 mg or 5 mL of tofacitinib citrate.
The results: The trial met its primary end point, which was statistically significant (P=0.0007). At week 44, the occurrence of disease flare in patients treated with tofacitinib citrate was 29% compared with 53% of patients treated with placebo.
Also at week 44, JIA ACR50/70 response rates and change from phase 2 baseline in CHAQ-DI scores were greater in patients who received tofacitinib citrate than in those who received placebo. The JIA ACR50 response rates were 66.7% vs. 47%, respectively, and the JIA ACR70 response rates were 54.3% vs. 37.1%, respectively. Also, the response rates for CHAQ-DI were 0.09 vs. 0.03 for tofacitinib compared with placebo. Statistical significance was not reported.
Additionally, time to disease flare was longer in patients treated with tofacitinib citrate than in patients who received placebo. These patients also had significantly fewer flares and improvements in signs and symptoms of disease and physical functioning.
Safety was similar between the study groups. The most common adverse effects were upper respiratory tract infections, nausea, vomiting, nasopharyngitis, fever and headache. No cases of death, opportunistic infection or tuberculosis occurred during the study.
Both formulations, the 5 mg tablet and 1 mg/mL oral solution of tofacitinib citrate, were approved to treat this patient population.
Prolonged-Release Tablet Approved
The EC also approved tofacitinib citrate in the form of a 11 mg prolonged-release tablet to treat adults with active PsA who do not tolerate methotrexate or for whom methotrexate or other DMARDs have proved inadequate. This once-daily, 11 mg tablet is an alternative treatment to the currently approved 5 mg tablet of tofacitinib citrate, which is taken twice daily.1
