The agency has approved nintedanib (Ofev) to slow the rate of pulmonary function decline in adult patients with interstitial lung disease associated with systemic sclerosis or scleroderma.
The FDA has approved the first treatment for patients with a rare type of lung disease. Late last week, the agency approved nintedanib (Ofev) to slow the rate of pulmonary function decline in adult patients with interstitial lung disease associated with systemic sclerosis or scleroderma (SSc-ILD).
Scleroderma, which impacts approximately 100,000 people in the United States, causes tissue throughout the body, including in the lungs, to thicken and scar. ILD is one of the most common manifestations of scleroderma, impacting nearly half of those with the disease. For patients with SSc-ILD, lung function declines over time, which can be debilitating and life threatening.
“Patients suffering from scleroderma need effective therapies, and the FDA supports the efforts of drug companies that are designing and conducting the clinical trials necessary to bring treatment options to scleroderma patients,” said Nikolay Nikolov, MD, associate director for Rheumatology of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, in a statement.
The effectiveness of nintedanib was demonstrated in a trial of 576 patients aged 20 to 79 years with the disease who were treated for at least 52 weeks; some were treated for up to 100 weeks. Compared with those taking placebo, patients who took nintedanib had less lung function decline, with the drug slowing the loss of pulmonary function by 44%.
The safety profile of the drug observed throughout the study was consistent with that known of the therapy. The most frequently reported serious adverse event was pneumonia (2.8% for nintedanib vs 0.3% for placebo). Approximately one-third of patients taking nintedanib had adverse reactions leading to dose reductions compared with 4% of those taking placebo.
“An approved anti-fibrotic medication for this condition is a scientific advancement in the care of patients living with this rare disease,” Kristin Highland, MD, a pulmonologist at the Cleveland Clinic, said in a statement. “The option to offer a new therapy is welcome news for doctors and their patients.”
Prescribing information for the drug includes warnings for patients with moderate or severe liver impairment, patients with elevated liver enzymes and drug-induced liver injury, and patients with gastrointestinal disorders. The FDA also warned that nintedanib could also cause embryo-fetal toxicity that can result in fetal harm, blood clots, bleeding, and gastrointestinal perforation.