The Food and Drug Administration’s (FDA) Arthritis Advisory Committee voted 10 to 7 in favor of approval of Ofev (nintedanib; Boehringer Ingelheim) for the treatment of systemic sclerosis associated with interstitial lung disease (ILD).
The recommendation was based on data from the phase 3 SENSCIS trial which evaluated the efficacy and safety of nintedanib in patients with systemic sclerosis and associated ILD (N=576); the primary end point of the study was the annual rate of decline in forced vital capacity (FVC) over a 52-week period.
Results showed that at Week 52, the adjusted annual rate of decline in FVC was lower in patients who received nintedanib (−52.4mL/year) compared with those who received placebo (−93.3mL/year), with a treatment difference of 41.0mL/year (95% CI: 2.9 to 79.0; P=.04). However, the key secondary end points (skin thickness as measured by the modified Rodnan Skin Score and quality of life as measured by St. George’s Respiratory Questionnaire) were not supportive of a direct benefit for nintedanib over placebo.
“We want to ensure that new products have a favorable benefit-risk assessment for patients,” the panel stated in meeting documents. “While the results for the primary end point were statistically significant based on the pre-specified analysis, the sensitivity analyses on missing data assumptions and responder analyses with various thresholds showed mixed results, mainly because the magnitude of the effect size was small.” The committee also noted that study findings showed a smaller treatment effect with nintedanib in patients on mycophenolate mofetil at baseline (treatment difference of 26.6mL/year) and no improvement in mortality.
With regard to safety, the most common adverse event was diarrhea (reported in 75.7% of the nintedanib group vs 31.6% of the placebo group).
Commenting on the vote, Thomas Seck, MD, senior vice president, Medicine and Regulatory Affairs, Boehringer Ingelheim said, “Based on the strength of the data presented, and the positive recommendation by the committee, we are hopeful that the FDA will approve nintedanib as a treatment option for patients with SSc-ILD. If approved, nintedanib would be the first treatment approved in the US for these patients.”
Although not bound by the committees’ recommendations, the FDA does take them into consideration when making decisions on approval.
Nintedanib, a kinase inhibitor, is currently indicated for the treatment of idiopathic pulmonary fibrosis.
For more information visit fda.gov.