Rare Daily Staff

The U.S. Food and Drug Administration has approved Boehringer Ingelheim’s Ofev capsules to slow the rate of decline in pulmonary function in adults with interstitial lung disease associated with systemic sclerosis or scleroderma (SSc-ILD).

It is the first FDA-approved treatment for this rare lung condition.

“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, associate director for Rheumatology of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research. “Nintedanib (Ofev) is now a treatment option to slow the rate of decline in pulmonary function in patients who have interstitial lung disease from scleroderma.”

Scleroderma is a rare disease that causes tissue throughout the body to thicken and scar. Interstitial lung disease or ILD is a condition affecting the interstitium, which is part of the lung’s structure, and is one of the most common disease manifestations of scleroderma. SSc-ILD is a progressive lung disease in which lung function declines over time, and it can be debilitating and life-threatening. 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. Approximately 100,000 individuals in the United States have scleroderma, and approximately half of scleroderma patients have SSc-ILD.

Nintedanib, trade name Ofev, was first approved by the FDA in 2014 for the treatment of adults with idiopathic pulmonary fibrosis. Its approval to treat SSc-ILD was based on a randomized, double-blind, placebo-controlled trial of 576 patients ages 20-79 with the disease. Patients received treatment for 52 weeks, with some patients treated up to 100 weeks. The primary test for efficacy measured the forced vital capacity, a measure of lung function defined as the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. Those who took Ofev had less lung function decline compared to those on placebo.

The overall safety profile was consistent with the known safety profile of the therapy. The most frequent serious adverse event reported in patients treated with Ofev was pneumonia, which occurred in 2.8 of treated patients versus 0.3 percent of those given a placebo.  Adverse reactions leading to permanent dose reductions were reported in 34 percent of Ofev-treated patients compared to 4 percent of placebo-treated patients. Diarrhea was the most frequent adverse reaction that led to permanent dose reduction in patients treated with Ofev.

The FDA granted Ofev Priority Review designation, which accelerates to six months the time it takes the FDA to take action on an application, and Orphan Drug designations, which provides incentives to assist and encourage the development of drugs for rare diseases.

X