New data from Boehringer Ingelheim support the potential use of nintedanib in children and adolescents with fibrosing interstitial lung disease

Ingelheim, Germany and Ridgefield, Conn, 5 September, 2022 – Today, Boehringer Ingelheim announced Phase III data from the InPedILD trial, which assessed the pharmacokinetics (dosing) and safety profile of nintedanib in children and adolescents between 6 and 17 years old with clinically significant fibrosing interstitial lung disease (ILD). The trial showed encouraging data for both primary endpoints and results were published in the European Respiratory Journal (ERJ) and presented at the European Respiratory Society International Congress (ERS) in Barcelona, Spain.¹

“Based on the nintedanib mode of action, preclinical evidence and the clinical benefit in adults, there was a compelling rationale for examining its effect in children living with interstitial lung disease,” said the coordinating investigator, Prof. Robin Deterding, M.D., Director of the Breathing Institute, Children's Hospital Colorado. “This trial supports its potential use as a treatment with an acceptable safety profile for children and adolescents, for whom no approved evidence-based therapies exist.”

The InPedILD results showed that the weight-based dosing regimen of nintedanib in children and adolescents with fibrosing ILD resulted in comparable exposure to that observed in adult patients with fibrosing ILD. In addition, nintedanib had an acceptable safety and tolerability profile with no new safety signals observed when compared to adult patients with idiopathic pulmonary fibrosis (IPF), other progressive fibrosing interstitial lung disease (PF-ILD), and systemic sclerosis-associated interstitial lung disease (SSc-ILD).¹ Based on these findings, regulatory applications will be submitted to the European Medicines Agency and U.S. Food and Drug Administration.

“While childhood interstitial lung diseases are very rare, their impact on children, teenagers and their loved ones can be devastating,” said Dr. Susanne Stowasser, Associate Head of Medicine Pulmonology at Boehringer Ingelheim. “The findings from InPedILD help meet the urgent need for well-characterized therapies for these children and adolescents living with ILD. These data further support Boehringer Ingelheim’s ongoing commitment to address unmet needs and advance research for people across all generations living with pulmonary fibrosis.”

Childhood interstitial lung disease (chILD) includes more than 200 rare disorders with debilitating symptoms that can include cough, difficulty breathing and rapid breathing.^2,3 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.² Pulmonary fibrosis within chILD is even less frequent, with no known global prevalence estimates, and no international studies prior to InPedILD.² 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.^3.4 There are no established diagnostic criteria and few management guidelines.^2,5,6 The current standard of care involves off-label use of treatments including steroids and steroid-sparing immunosuppressants, which have known adverse events and limited evidence for their use.²

About the trial 

• InPedILD (NCT04093024) is a Phase III double-blind, randomized, placebo-controlled trial assessing dose exposure and safety of nintedanib on top of standard of care for 24 weeks, followed by open-label treatment with nintedanib of variable duration in children and adolescents aged 6-17 years with clinically significant fibrosing ILD. It is one of the first randomized controlled clinical trials focused on childhood interstitial lung diseases.
• The pharmacokinetic results demonstrated that the exposure to nintedanib in children was within the variability of that observed in adults treated with the approved dose, supporting the use of a weight-based dosing regimen in the pediatric population. 
• The safety endpoint was based on the proportion of patients with treatment-emergent adverse events at Week 24. As in adults, the most common adverse event associated with nintedanib in the InPedILD trial was diarrhea. All reported diarrhea adverse effects could be resolved without premature discontinuation of trial medication.

About Nintedanib 

Nintedanib is a tyrosine kinase inhibitor targeting key receptors involved in signaling pathways that lead to pulmonary fibrosis. It is approved in more than 80 countries including the European Union, U.S. Brazil, Canada and Japan, for the treatment of patients living with idiopathic pulmonary fibrosis (IPF), systemic sclerosis-associated interstitial lung disease (SSc-ILD) and other chronic fibrosing ILDs with a progressive phenotype.  

About Boehringer Ingelheim

Boehringer Ingelheim is working on breakthrough therapies that transform lives, today and for generations to come. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. More than 52,000 employees serve over 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. Learn more at www.boehringer-ingelheim.com

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References

1. Deterding R. et al. Nintedanib in children and adolescents with fibrosing interstitial lung diseases. Eur Respir J. 2022

2. Deterding R. et al. Study design of a randomised, placebo controlled trial of nintedanib in children and adolescents with fibrosing interstitial lung disease. ERJ Open Res. 2021 Jun 21;7(2):00805-2020

3. Deterding R. et al. Approaching Clinical Trials in Childhood Interstitial Lung Disease and Pediatric Pulmonary Fibrosis. Am J Respir Crit Care Med. 2019 Nov 15;200(10):1219-1227.

4. Cunningham S. et al. Children's interstitial and diffuse lung disease. Lancet Child Adolesc Health. 2019; 10.1016/S2352-4642(19)30117-8. 

5. Kurland et al. An official American Thoracic Society clinical practice guideline. Am J Respir Crit Care Med. 2013 Aug 1;188(3):376-94.

6. Bush et al. European protocols for the diagnosis and initial treatment of interstitial lung disease in children. Thorax. 2015 Nov;70(11):1078-84