FDA Accepts sNDA for Ofev in Children with Rare Lung Disease

The FDA has accepted Boehringer Ingelheim’s supplemental new drug application (sNDA) for Ofev (nintedanib), to treat children and adolescents between 6 to 17 years old with fibrosing interstitial lung disease (ILD). A decision is expected in the fourth quarter of this year.

Interstitial lung disease is rare condition that includes more than 200 disorders, with about 30 disorders that effect children. There are less than 10,000 estimated cases in the United States in children, according to the Children’s Interstitial Lung Disease Foundation. Pulmonary fibrosis is a lung disease that occurs when lung tissue becomes damaged and scarred, making it difficult to breath.

Ofev is a tyrosine kinase inhibitor that works by inhibiting pathways involved in pulmonary fibrosis. It is currently approved to treat adults with idiopathic pulmonary fibrosis, chronic fibrosing interstitial lung disease and interstitial lung disease associated with systemic sclerosis.

The cost is about $12,496 for a supply of 60 capsules. A spokesperson said the price is similar to other therapies for rare/orphan diseases approved by the FDA. Boehringer Ingelheim offers a number of programs designed to support patients, including copay assistance for eligible patients and a patient support program.

For the children's indication, dosing would be weight-based, and the spokesperson would not comment on the price for a children's dose.

Robin Deterding, M.D.

“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,” Robin Deterding, M.D., director of the Breathing Institute, Children’s Hospital Colorado, said in a press release. “If approved, Ofev would be the first and only approved treatment for children and adolescents aged 6 to 17 years old with fibrosing interstitial lung disease, representing an important step forward for these patients.”

The sNDA is based on results from the InPedILD phase 3 trial, which evaluated dose-exposure and safety of Ofev on top of standard of care. In children and adolescents with fibrosing ILD, a weight-based dosing regimen resulted in exposure of Ofev similar to adults and with a similar safety profile. The most common adverse event associated with treatment was diarrhea. The complete results were published last year in the European Respiratory Journal (ERJ) and presented at the European Respiratory Society (ERS) International Congress in September 2022.