Ofev for fibrosing ILDs in children as young as 6 under FDA review
If approved, Ofev would become first therapy for children with fibrosing ILDs
The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking the approval of Ofev (nintedanib) for children ages 6 to 17 with fibrosing interstitial lung diseases (ILDs).
If approved, Ofev would become the first and only therapy available to treat children with fibrosing ILDs, according to its manufacturer Boehringer Ingelheim.
“We are pleased that the FDA recognizes the unmet need for this patient population, and we look forward to working with the FDA to potentially bring forward the first approved option as quickly as possible,” Craig Conoscenti, MD, executive director and therapeutic area head, respiratory IPF/ILD, clinical development and medical affairs at Boehringer Ingelheim, said in a company press release.
ILDs can severely affect health and quality of life for children
ILDs encompass more than 200 lung disorders characterized by inflammation and scarring (fibrosis). While these conditions rarely develop in children, they can have a severe impact on their health and quality of life.
“With a long and complicated journey to diagnosis and no approved therapies, childhood interstitial lung diseases can place a significant burden on patients and their families,” said Robin Deterding, MD, director of the Breathing Institute at Children’s Hospital Colorado.
Pulmonary fibrosis can develop in children with ILDs and is associated with worse outcomes. The lungs normally need to be stretchy like a balloon to fill up with air, but fibrosis makes lung tissue stiffer so it’s harder to take in air.
Ofev is an oral anti-fibrotic medication that works by blocking certain signaling molecules that drive lung fibrosis. The therapy is currently approved for managing several lung disorders in adults, including idiopathic pulmonary fibrosis, scleroderma-associated ILD, and progressive fibrosing ILDs.
“If approved, Ofev would be the first and only approved treatment for children and adolescents aged 6-17 years old with fibrosing interstitial lung disease, representing an important step forward for these patients,” Deterding said.
FDA application based on data from Phase 3 InPedILD clinical trial
Boehringer Ingelheim’s supplemental new drug application is based on data from a Phase 3 clinical trial called InPedILD (NCT04093024). The study enrolled 39 children with fibrosing ILDs who were treated with Ofev or a placebo for about half a year.
Results, published last year, showed that the therapy was generally well tolerated in these young children, and showed that the weight-based dosing program used resulted in levels of the medication in the body similar to those seen in treated adults.
“We remain committed to building on our heritage in respiratory innovation with an aim to provide life-changing treatments for patients living with pulmonary fibrosis, including those living with childhood interstitial lung disease,” Conoscenti said.