The U.S. Food and Drug Administration (FDA) recently approved a treatment for a rare form of lung disease, according to an agency press release.
The treatment involves taking Ofev (Nintedanib) capsules to slow the rate of decline in pulmonary function in adults with interstitial lung disease associated with systemic sclerosis or scleroderma (SSc-ILD). Dr. James Kiley, director of the Division of Lung Diseases at the National Heart, Lung and Blood Institute, weighed in on the first FDA-approved treatment for this rare condition.
"Systemic sclerosis-associated interstitial lung disease has a variable course, but many patients have disease progression, with a decline in FVC, which is a measure of lung function," Kiley told FDA Health News. "The results of this randomized study suggest that Nintedanib reduces the rate of FVC decline compared to placebo. However, there was no perceived benefit for other aspects of systemic sclerosis. The results of this study support the use of Nintedanib in SSc-ILD."
Nikolay Nikolov, M.D., associate director for rheumatology at the Division of Pulmonary, Allergy and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, expressed the importance of developing new treatment options for SSc-ILD.
"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," Nikolov said in the press release. "Nintedanib is now a treatment option to slow the rate of decline in pulmonary function in patients who have interstitial lung disease from scleroderma."
Kiley suggested that Nintedanib could potentially be used in collaboration with previous treatments for this rare disease.
"An NHLBI-funded Scleroderma Lung Study II trial showed that Mycophenolate mofetil (MMF) had a better safety profile and comparable efficacy to cyclophosphamide in patients with SSc-ILD,’’ Kiley said in the release. "Notably, nearly 50 percent of patients enrolled in the current clinical trial were taking a stable dose of MMF at baseline. The authors of the current study suggest a benefit of MMF on lung-function decline. This might suggest that use of MMF and Nintedanib as combination therapy could be beneficial, but this has not been proven in a randomized clinical trial."
So what is scleroderma exactly? The disease causes tissue throughout the body, including the lungs, to thicken and scar. Approximately 100,000 individuals in the United States have scleroderma, and approximately half of scleroderma patients have SSc-ILD.
"SSc-ILD is a progressive lung disease in which lung function declines over time, and it can be debilitating and life-threatening," the press release states. "ILD is the leading cause of death among people with scleroderma, typically resulting from a loss of pulmonary function that occurs when the lungs cannot supply enough oxygen to the heart."
The FDA granted the approval of Ofev to treat SSc-ILD to Boehringer Ingelheim Pharmaceuticals Inc.
