Nintedanib expanded access program is announced for children and adolescents with fibrosing interstitial lung disease

Nintedanib is approved by the FDA for the treatment of adults with idiopathic pulmonary fibrosis (IPF), chronic fibrosing ILDs with a progressive phenotype, and to slow the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated ILD (SSc-ILD). Nintedanib has not been approved for use in children and adolescents.

Expanded access to nintedanib would provide an investigational treatment for children and adolescents who otherwise have no approved therapies for this illness.

“With no established diagnostic criteria, and no approved therapies, childhood interstitial lung diseases can place a significant burden on patients and their loved ones,” said Craig Conoscenti, M.D., FCCP, ATSF, Executive Director/Therapeutic Area Head, Respiratory IPF/ILD, Clinical Development and Medical Affairs, Boehringer Ingelheim. “Given the debilitating and progressive nature of this rare disease, Boehringer Ingelheim remains committed to making nintedanib accessible to children and adolescents who are unable to take part in our ongoing clinical trial program.”

EAPs are intended to allow patient access to an investigational medicine for a serious or immediately life-threatening disease or condition where no comparable or satisfactory alternative therapy exists and when the potential benefit of the medicine justifies the potential risks and those risks are not unreasonable in the context of the disease or condition being treated.

Childhood interstitial lung disease (chILD) includes more than 200 rare disorders with debilitating symptoms that can include cough, difficulty breathing and rapid breathing. Its exact prevalence is unknown, but it can be considered very rare with a reported incidence ranging from 1.5 to 3.8 per million. Pulmonary fibrosis within chILD is even less frequent, with no known global prevalence estimates, and no international studies prior to InPedILD. chILD is associated with significant mortality and morbidity. When their condition deteriorates, many pediatric patients will need oxygen to go about their daily lives and require lung transplants. There are no established diagnostic criteria and few management guidelines. The current standard of care involves off-label use of treatments including steroids and steroid-sparing immunosuppressants.