Boehringer Ingelheim’s Ofev has become the first drug approved by the FDA for a lung disease that afflicts patients with systemic sclerosis, a rare autoimmune disease.
Ofev (nintedanib) – which has been approved for several years as a treatment for idiopathic pulmonary fibrosis (IPF) – was shown in trials to slow the rate of lung function decline in systemic sclerosis-associated interstitial lung disease (SSc-ILD).
Scleroderma is a rare disease that causes tissue throughout the body, including the lungs and other organs, to thicken and scar. It affects around 100,000 people in the US and 2.5m worldwide, with around 80% of all patients going on to develop lung disease, the leading cause of death in SSc.
The orally-acting drug has also been filed for SSc-ILD in Europe with a verdict expected early next year.
It was recommended for approval by an FDA advisory committee in July by a narrow margin, 10 votes to seven, with some panellists raising concerns about a risk of pneumonia in the trial data. Now it has been approved after a priority review by the US regulator, reflecting the lack of therapies available for SSc-ILD.
The FDA approval comes on the back of the 576-patient SENSCIS trial reported earlier this year, which found that Ofev – a triple angiokinase inhibitor that simultaneously inhibits VEGF, PDGF and FGF receptors – achieved a 44% reduction in the rate of decline of lung function over 52 weeks compared to placebo.
The trial tested patients’ forced vital capacity (FVC), 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.
“An approved anti-fibrotic medication for this condition is a major scientific advancement in the care of patients living with this rare disease,” said Kristin Highland, a pulmonologist with the Cleveland Clinic and a SENSCIS investigator.
“The option to offer a new therapy is welcome news for doctors and their patients,” she added.
Ofev is already a blockbuster seller for Boehringer, growing almost 29% to reach €1.1bn ($1.2bn) last year from its use in IPF, which also affects around 100,000 Americans according to National Institutes of Health (NIH) estimates.
Ofev’s active ingredient nintedanib is also sold as Vargatef for some types of non-small cell lung cancer (NSCLC).