The U.S. Food and Drug Administration (FDA) has approved Ofev (nintedanib) as the first therapy to slow lung function decline in people with interstitial lung disease (ILD) associated with systemic sclerosis (SSc-ILD), Boehringer Ingelheim announced.
SSc affects multiple systems in the body, causing progressive, widespread fibrosis (tissue scarring). Within the first three years after diagnosis, 25% of scleroderma patients develop scars in the lungs, which can lead to ILD, the leading cause of death among these patients.
ILD is a group of lung disorders in which the tissue in and around the lung air sacs — called the interstitium — becomes inflamed and scarred, affecting breathing and impairing lungs’ ability to transfer oxygen to the bloodstream.
Ofev, marketed by Boehringer Ingelheim, is an approved treatment for idiopathic pulmonary fibrosis — the most common ILD — in more than 70 countries. It works by blocking a group of growth factor receptors involved in lung fibrosis.
“This is the first FDA-approved therapy to slow the rate of decline in pulmonary function for systemic sclerosis-associated interstitial lung disease, and provides hope to patients and their loved ones facing this devastating disease,” Thomas Seck, MD, Boehringer Ingelheim’s medicine & regulatory affairs senior vice president, said in a press release.
The recent approval was based on results of the randomized, double-blind, placebo-controlled Phase 3 SENSCIS trial (NCT02597933), which evaluated the safety and effectiveness of Ofev in 576 patients with SSc-ILD.
Patients were recruited at 194 trial sites across 32 countries, and randomly assigned to receive either 150 mg of Ofev or a placebo twice daily for at least a year (52 weeks).
The trial’s primary goal was assessing the annual rate of decline in lung forced vital capacity (FVC) — a measure of lung function, defined as the amount of air a person can forcibly exhale after taking the deepest breath possible.
Results showed that after one year of treatment, patients receiving Ofev had a significantly slower decline in lung function (44% less decline in FVC) compared with those receiving a placebo.
Ofev’s overall safety profile was consistent with the therapy’s known safety profile, with diarrhea being the most frequently reported adverse event. Nausea, vomiting, fatigue, and weight loss were also among the most common adverse events associated with Ofev treatment.
“An approved anti-fibrotic medication for this condition is a scientific advancement in the care of patients living with this rare disease,” said Kristin Highland, MD, a Cleveland Clinic pulmonologist.
In 2016, both the FDA and the European Medicines Agency (EMA) designated Ofev as an orphan drug for the treatment of scleroderma, as well as associated ILD, a status that provides incentives to assist and promote the development of therapies for rare diseases.
Earlier this year, Ofev was granted priority review by the FDA, under which the agency’s goal is to take action on an application within six months.
“When interstitial lung disease occurs in systemic sclerosis, the consequences can be severe. The availability of a new therapy for patients with this rare, chronic condition is truly exciting news for our community and at the core of the mission of the Scleroderma Research Foundation to improve the lives of patients,” said Luke Evnin, PhD, a scleroderma patient and chairman of the Scleroderma Research Foundation.
Boehringer Ingelheim is currently conducting an open-label extension study (NCT03313180) to evaluate the long-term effects of Ofev in people with SSc-ILD.
The company has a patient support program to help those prescribed Ofev. The OPEN DOORS program provides a broad range of nursing, social resources, and financial support services.