Ofev (nintedanib) is a treatment for interstitial lung disease caused by systemic scleroderma or scleroderma that affects internal organs and tissues. Ofev is manufactured by Boehringer Ingelheim and has been approved by the U.S. Food and Drug Administration (FDA) to slow the decline of lung function in patients with interstitial lung disease caused by systemic scleroderma.
What is scleroderma?
Scleroderma is a disease that affects the connective tissue – the tissue that supports joints and gives structure to organs. It’s an autoimmune disease where the immune system is overactivated as if the connective tissue is under attack. Cells called fibroblasts then start to produce a connective tissue protein called collagen, which forms the basis of scar tissue.
In normal healing, once tissue has scarred, there is no need to continue producing collagen.
In scleroderma, however, the fibroblasts continue producing collagen as if they’re responding to an active threat. This leads to the overproduction of fibrous connective tissue (scarring) that can interfere with organ function. The scarring can be especially dangerous in the lungs, where scar tissue prevents the lungs from inflating properly and impedes the transport of oxygen from the lungs to the blood.
About 25% of patients with systemic scleroderma develop interstitial lung disease, which refers to a group of disorders in which tissues and spaces around the lungs’ air sacs, known as the interstitium, become inflamed and damaged.
How does Ofev work?
Ofev contains a small molecule (nintedanib), which inhibits enzymes called tyrosine kinases. It works by targeting proteins called growth factor receptors, known to be involved in interstitial lung disease. Ofev targets and blocks specific receptors called the platelet-derived growth factor receptor, fibroblast growth factor receptor, and vascular endothelial growth factor receptor, which are produced in high levels — thereby initiating the inflammation process — in patients with interstitial lung disease.
By blocking these receptors, Ofev reduces the signaling pathways involved in scarring, thereby slowing the decline of lung function in patients with interstitial lung disease.
Ofev in clinical trials
The safety and efficacy of Ofev — under its chemical name nintedanib — in treating scleroderma-related pulmonary fibrosis was tested in a Phase 3 clinical trial (NCT02597933) called SENSCIS.
In this global, randomized, and double-blind trial, 576 scleroderma patients with associated fibrotic lung disease were given either nintedanib capsules at a dose of 150 mg twice a day or a placebo twice a day for at least 52 weeks.
Researchers analyzed the medicine’s effectiveness in slowing disease progression by assessing the rate of lung function decline after one year of treatment. Specifically, they measured the patients’ forced vital capacity (FVC) or the amount of air that they can forcibly exhale from their lungs after taking the deepest possible breath. Secondary trial goals included changes in tissue thickness and health-related quality of life.
The results of the SENSCIS trial formed the basis of the FDA’s approval of the treatment. They showed that following one year of treatment, patients who received Ofev demonstrated a significantly slower decline in lung function (44% less decline in FVC), compared to the control group receiving the placebo.
The safety of the treatment was consistent with previous trials. No serious adverse events were seen. Side effects (occurring in 5% or more of patients) included diarrhea, nausea, vomiting, skin ulcer, abdominal pain, liver enzyme elevation, weight loss, fatigue, decreased appetite, headache, fever, and high blood pressure.
About 400 patients in the Phase 3 trial are being invited to enroll in a long-term extension study (NCT03313180), which will evaluate the safety of nintedanib by looking at adverse events that occur up to 34 months after treatment. This study is open-label, meaning that all patients will be made aware that they’re taking nintedanib orally twice a day. The trial is expected to end in July 2021.
Common side effects of Ofev include diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, headache, weight loss, and high blood pressure.
Blood tests to assess liver function are frequently needed for those taking Ofev to verify that the medicine is not harming the liver. Women of childbearing age should not take Ofev, as it can cause birth defects and harm the fetus.
Last updated: Sept. 17, 2019
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