CHMP recommends approval of OFEV® in children and adolescents for the treatment of a range of interstitial lung diseases (ILDs)

• OFEV® would become the first and only approved treatment for pediatric patients with clinically significant, progressive fibrosing ILDs and for pediatric patients with SSc-ILD.
• The CHMP positive opinion is primarily based on the InPedILD Phase III clinical trial, which showed that the weight-based dosing regimen of OFEV® in children and adolescents with fibrosing ILD resulted in comparable exposure to that observed in adult patients with fibrosing ILD.¹
• The InPedILD trial in children and adolescents with fibrosing ILD showed that nintedanib had an acceptable safety profile.² It was not powered for efficacy but in an exploratory fashion assessed changes in Forced Vital Capacity [mL] (FVC) from baseline, which were also considered in a Bayesian extrapolation analysis estimating the effect of nintedanib in this patient population.^2,3

Why it matters

• Childhood Interstitial Lung Disease (chILD) can occur in more than 200 rare disorders, affecting infants, children and adolescents, with debilitating symptoms that can include cough, difficulty breathing and rapid breathing.^1,4 Many children need to go on oxygen to go about their daily lives and require lung transplants as their condition deteriorates.⁵ With no established diagnostic criteria or approved therapies, chILD can be difficult to diagnose and manage.^4,6,7
• There has been an urgent need to develop safe and efficacious therapies for chILD and associated fibrosing ILD. 
• chILD is considered a rare disease with a newly reported incidence of around 4 to 8 cases/million and prevalence of around 45 cases/million but its exact global prevalence is unknown.^{4,8,9,10,11,12,13} Pulmonary fibrosis associated with chILD is even less frequent, ultra-rare, estimated to occur in 2% to 7% of patients with chILD.^14,15
• Systemic Sclerosis (SSc) is less common in the pediatric population compared with the adult population. Juvenile Systemic Sclerosis (jSSc) is a rare autoimmune connective tissue disease with a reported incidence rate of 0.27-2.9 per million children person-years (<16 years old) and a prevalence rate of ~4 per million children (<16 years old).^{16,17,18,19,20,21} The proportion of pulmonary fibrosis in patients with juvenile SSc (~40%) is approximately in the same range as in adult patients with SSc.²²

“Children battling these rare conditions are often overlooked within clinical trials due to the complexities of pediatric dosing and additional steps in the participant approval process, leaving a critical gap in our understanding and treatment options,” said Martin Beck, Head of Therapeutic Area Inflammation. “The CHMP recommendation is a significant step forward in our commitment to addressing the unmet needs of all those affected by pulmonary fibrosis. We’re fighting for these children and their families so they can have a chance at a brighter future.”

About the trial

• InPedILD (NCT04093024) is a Phase III double-blind, randomized, placebo-controlled trial assessing dose exposure and safety of OFEV® on top of standard of care for 24 weeks, followed by open-label treatment with OFEV® 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 OFEV® 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 OFEV® in the InPedILD trial was diarrhea. All reported diarrhea adverse effects could be resolved without premature discontinuation of trial medication.
• Additional information about the trial can be found here: New data from Boehringer Ingelheim support the potential use of nintedanib in children and adolescents with fibrosing interstitial lung disease.

About OFEV®

OFEV® 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 in many members of 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.²³

About Boehringer Ingelheim

Boehringer Ingelheim is a biopharmaceutical company active in both human and animal health. As one of the industry’s top investors in research and development, the company focuses on developing innovative therapies that can improve and extend lives in areas of high unmet medical need. Independent since its foundation in 1885, Boehringer takes a long-term perspective, embedding sustainability along the entire value chain. More than 53,500 employees serve over 130 markets to build a healthier, more sustainable, and equitable tomorrow. Learn more at www.boehringer-ingelheim.com.

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