FDA OKs Nintedanib (Ofev) for Rare Lung Disease

Megan Brooks

September 09, 2019

The US Food and Drug Administration (FDA) has approved nintedanib (Ofev, Boehringer Ingelheim) capsules for slowing the rate of decline in pulmonary function in adults with systemic sclerosis–associated interstitial lung disease (SSc-ILD).

Nintedanib is the first FDA-approved treatment for this rare lung condition. The FDA's Arthritis Advisory Committee recommended approving nintedanib for SSc-ILD in July, as reported by Medscape Medical News.

Nintedanib is an oral triple angiokinase inhibitor that simultaneously inhibits signaling pathways of vascular endothelial growth factor receptors 1–3, platelet-derived growth factor receptors, and fibroblast growth factor receptors 1–3.

It was first approved in 2014 for a related disease, idiopathic pulmonary fibrosis.

SSc-ILD is a rare connective tissue disorder that affects about 100,000 people in the United States. Patients with SSc-ILD develop widespread fibrosis and vascular abnormalities in the skin, joints, and internal organs, such as the esophagus, lower gastrointestinal tract, heart, lungs, and kidneys. They also have an increased risk for morbidity and mortality.

"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," Nikolay Nikolov, MD, of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA's Center for Drug Evaluation and Research, said in a news 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," said Nikolov.

The efficacy of nintedanib for SSc-ILD was demonstrated in a randomized, double-blind, placebo-controlled trial of 576 patients aged 20 to 79 years who had the disease. Patients received treatment for 52 weeks; some patients were treated up to 100 weeks.

Results showed that, compared to patients who received placebo, those who took nintedanib experienced significantly less decline in lung function, as assessed on the basis of forced vital capacity, the FDA said.

The overall safety profile of nintedanib in SSc-ILD was consistent with the known safety profile of the drug. Common side effects of nintedanib include diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, headache, weight loss, and hypertension.

The most common serious adverse event reported in patients taking nintedanib was pneumonia (2.8%). Adverse reactions that led to permanent dose reductions were reported in 34% of nintedanib-treated patients, compared to 4% of placebo-treated patients.

The prescribing information for nintedanib includes warnings for patients with moderate or severe liver impairment, those with elevated liver enzyme levels and drug-induced liver injury, and those with gastrointestinal disorders.

The drug may cause embryo-fetal toxicity that can result in fetal harm, arterial thromboembolic events, bleeding, and gastrointestinal perforation. P-glycoprotein and CYP3A4 inhibitors may increase nintedanib exposure, and patients taking these inhibitors should be closely monitored for tolerability of the drug, the FDA said.

Nintedanib for SSc-ILD had orphan drug status and priority review designation.

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