FDA accepts sNDA for nintedanib for treatment of fibrosing interstitial lung disease

FDA accepts sNDA for nintedanib for treatment of fibrosing interstitial lung disease | Image Credit: © yodiyim - © yodiyim - stock.adobe.com.

This week, the FDA accepted a supplemental new drug application (sNDA) for nintedanib (OFEV; Boehringer Ingelheim) for the investigational treatment of fibrosing interstitial lung disease (ILD) in patients aged 6 to 17 years, Boehringer Ingelheim announced in a press release. Should the FDA approve nintedanib, it would become the first and only treatment approved for this patient population.¹

Over 200 disorders are included in rare childhood ILD, which has a reported incidence in 1.6 to 46 million children, though the exact prevalence is not known. Further, according to Boehringer Ingelheim, pulmonary fibrosis within childhood ILD has no known prevalence estimates on a global scale but is less frequent. Currently, no diagnostic criteria are established, and few management guidelines have been put in place.¹

The sNDA filing with the FDA follows the InPedILD phase 3 trial (NCT04093024), a double-blind, randomized, placebo-controlled trial that assessed dose exposure and safety of nintedanib.¹ An open-label treatment of variable duration with nintedanib was followed in children and adolescents with significant fibrosing ILD, aged 6 to 17 years.¹ Results were published in the European Respiratory Journal, and presented at the European Respiratory Society International Congress in September 2022.¹

According to published results, the primary outcomes were determining the dose of nintedanib that would result in an exposure comparable to adults, and to assess its safety in patients with clinically significant fibrosing ILD.² These endpoints were dose exposure at week 2 of nintedanib treatment and the proportion of patients with “treatment-emergent adverse events at week 24.”¹

Thirty-nine patients in the 6-to-17-year age group were treated with nintedanib or placebo.1 There were 12 patients aged 6 to 11 years and 27 patients aged 12 to 17 years. Overall, 26 were treated with nintedanib and 13 with placebo.¹ According to the authors, the results “demonstrated that nintedanib had an acceptable safety profile in this patient population.”² As with adults, diarrhea was the most common adverse event associated with nintedanib treatment, but “the proportion of children who experienced [diarrhea] was lower than that observed in adults,” and no discontinuations resulting from diarrhea were observed during the double-blind portion.²

Currently, nintedanib is a prescription medicine used to treat idiopathic pulmonary fibrosis (IPF) in adults, long-lasting ILD with lung fibrosis that continues to worsen in adults, and to “slow the rate of decline in lung function in adults with systemic sclerosis-associated ILD.” According to Boehringer Ingelheim, nintedanib could cause birth defects, harm, or death to an unborn baby. Women should not become pregnant while taking nintedanib, and those that can become pregnant should take a pregnancy test before treatment. “Highly effective” birth control at start of nintedanib treatment, during treatment, and at least 3 months after treatment should be used.¹

References:

1. US FDA accepts supplemental new drug application for OFEV (nintedanib) for children and adolescents aged 6-17 years old with fibrosing interstitial lung disease. Boehringer Ingelheim. Press release. July 25, 2023. Accessed July 27, 2023. https://www.boehringer-ingelheim.com/us/press-releases/us-fda-accepts-snda-children-adolescents
2. Deterding R, Young LR, DeBoer EM, et al. Nintedanib in children and adolescents with fibrosing interstitial lung diseases. European Respiratory Journal. Published online January 1, 2022. doi:10.1183/13993003.01512-2022