WASHINGTON -- Nintedanib (Ofev) may be marketed as a treatment for systemic sclerosis-associated interstitial lung disease (SSc-ILD), the FDA said Friday.
Previously approved to treat idiopathic pulmonary fibrosis, nintedanib becomes the first treatment specifically indicated for SSc-ILD, for which few options are available.
"Patients suffering from scleroderma [another name for systemic sclerosis] 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. "Nintedanib is now a treatment option to slow the rate of decline in pulmonary function in patients who have interstitial lung disease from scleroderma."
The FDA cited results from a 576-patient trial as key to making the drug approvable. In it, patients taking nintedanib still showed gradual deterioration in lung function, but at a 44% slower rate than those assigned to placebo.
In approving the drug, the FDA decided the drug's benefits outweighed some significant safety issues. At a July meeting, seven of the agency's 17 Arthritis Advisory Committee members voted against recommending approval on account of nintedanib's adverse effects, including pneumonia risk (seen in 2.8% of patients receiving the drug vs 0.3% of those on placebo) and possibly immunosuppression. Dose reductions in the clinical program were required in one-third of nintedanib patients but were rare with placebo.
But those voting in favor of approval said it was more important to have a drug effective against SSc-ILD, which is the leading cause of death in scleroderma.
Although committee members recommended post-marketing studies to examine alternative dosing regimens -- it was administered orally at 150 mg twice daily in the main trial -- the FDA's approval announcement did not indicate that it would require drugmaker Boehringer Ingelheim to conduct such studies.