• InPedILD Phase III trial showed encouraging results for both primary endpoints. Full data were published in the European Respiratory Journal and presented at the European Respiratory Society International Congress in Barcelona, Spain
• If approved, nintedanib would become the first approved treatment for pediatric patients with fibrosing interstitial lung disease, addressing a high unmet need
• Findings supplement the long-term and established clinical efficacy of nintedanib in adults, supporting Boehringer Ingelheim's leadership in pulmonary fibrosis and determination to transform lives for generations

RIDGEFIELD, Conn. and INGELHEIM, Germany, Sept. 5, 2022 /PRNewswire/ -- Today, Boehringer Ingelheim announced Phase III data from the InPedILD trial, which assessed the pharmacokinetics (dosing) and safety profile of nintedanib (OFEV^®) 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 (ERS) International Congress in Barcelona, Spain.

"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). 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. 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, which have known adverse events and limited evidence for their use.

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 OFEV

OFEV is already approved in the U.S. and more than 80 countries for the treatment of patients living with IPF. In September 2019, OFEV was approved in the U.S. to slow the rate of decline in pulmonary function in patients with SSc-ILD, and then in March 2020 to treat patients with chronic fibrosing ILDs with a progressive phenotype.

What is OFEV? 

• OFEV is a prescription medicine used: 
  • to treat adults with a lung disease called idiopathic pulmonary fibrosis (IPF). 
  • to treat adults with a long lasting (chronic) interstitial lung disease in which lung fibrosis continues to worsen (progress).
  • to slow the rate of decline in lung function in adults with systemic sclerosis-associated interstitial lung disease (SSc-ILD) (also known as scleroderma-associated ILD). 
• It is not known if OFEV is safe and effective in children. 

Important Safety Information 

What is the most important information I should know about OFEV? 

OFEV can cause harm, birth defects, or death to an unborn baby. Women should not become pregnant while taking OFEV. Women who are able to become pregnant should have a pregnancy test before starting treatment and should use highly effective birth control at the start of treatment, during treatment, and for at least 3 months after your last dose. Talk with your doctor about what birth control method is right for you during this time. Birth control pills may not work as well in women having vomiting, diarrhea, or other problems reducing the drug absorption. If you have any of these problems, talk with your doctor about what highly effective birth control method is right for you. If you become pregnant or think you are pregnant while taking OFEV, tell your doctor right away. 

What should I tell my doctor before using OFEV? 

Before you take OFEV, tell your doctor about all of your medical conditions, including if you have: 

• liver problems.
• heart problems.
• a history of blood clots.
• a bleeding problem or a family history of a bleeding problem.
• had recent surgery in your stomach (abdominal) area.

 Tell your doctor if you: 

• are pregnant or plan to become pregnant.
• are breastfeeding or plan to breastfeed. It is not known if OFEV passes into your breast milk.
            You should not breastfeed while taking OFEV.
• are a smoker. You should stop smoking prior to taking OFEV and avoid smoking during treatment.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements such as St. John's wort. 

What are the possible side effects of OFEV? 

OFEV may cause serious side effects. 

TELL YOUR DOCTOR RIGHT AWAY if you are experiencing any side effects, including: 

• Liver problems. Unexplained symptoms may include yellowing of your skin or the white part of your eyes (jaundice), dark or brown (tea-colored) urine, pain on the upper right side of your stomach area (abdomen), bleeding or bruising more easily than normal, feeling tired, or loss of appetite. Your doctor will do blood tests to check how well your liver is working before starting and during your treatment with OFEV.
• Diarrhea, nausea, and vomiting. Your doctor may recommend that you drink fluids or take medicine to treat these side effects. Tell your doctor if you have these symptoms, if they do not go away, or get worse, and if you are taking over-the-counter laxatives, stool softeners, and other medicines or dietary supplements.
• Heart attack. Symptoms of a heart problem may include chest pain or pressure, pain in your arms, back, neck, or jaw, or shortness of breath.
• Stroke. Symptoms of a stroke may include numbness or weakness on one side of your body, trouble talking, headache, or dizziness.
• Bleeding problems. OFEV may increase your chances of having bleeding problems. Tell your doctor if you have unusual bleeding, bruising, wounds that do not heal, and/or if you are taking a blood thinner, including prescription blood thinners and over-the-counter aspirin.
• Tear in your stomach or intestinal wall (perforation). OFEV may increase your chances of having a tear in your stomach or intestinal wall. Tell your doctor if you have pain or swelling in your stomach area.
• Increased protein in your urine (proteinuria). OFEV may increase your chances of having protein in your urine. Tell your doctor if you have any signs and symptoms of protein in the urine such as foamy urine, swelling, including in your hands, arms, legs, or feet, or sudden weight gain.

The most common side effects of OFEV are diarrhea, nausea, stomach pain, vomiting, liver problems, decreased appetite, headache, weight loss, and high blood pressure. 

These are not all the possible side effects of OFEV. For more information, ask your doctor or pharmacist. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. 

Please see full Prescribing Information, including Patient Information.

CL-OF-100056 01.18.2022

About Boehringer Ingelheim

Boehringer Ingelheim is working on breakthrough therapies that improve the lives of humans and animals. 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. Around 52,000 employees serve more than 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing.  

Learn more at www.boehringer-ingelheim.us

Contact:

Boehringer Ingelheim Pharmaceuticals, Inc.
Name: Kerry Wycislo
Human Pharma Communications, Corporate Affairs
Phone: 203-561-2829
Email: kerry.wycislo@boehringer-ingelheim.com  

SOURCE Boehringer Ingelheim Pharmaceuticals