FDA Grants nintedanib Breakthrough Therapy Designation for Chronic Fibrosing ILDs with a Progressive Phenotype

Ridgefield, Conn., October 10, 2019 – Boehringer Ingelheim announced today that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to nintedanib, which is currently under FDA review for the treatment of people with chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype. Regulatory applications have been submitted to other regulatory bodies, including the European Medicines Agency.

The Breakthrough Therapy Designation was supported by results from the Phase III INBUILD® study – the first clinical trial of these ILDs that met its primary endpoint and showed nintedanib slowed the rate of ILD progression in patients with a broad range of progressive fibrosing interstitial lung diseases other than idiopathic pulmonary fibrosis (IPF).¹ The results were recently presented at the European Respiratory Society (ERS) International Congress in Madrid and published in the New England Journal of Medicine.

Interstitial lung diseases encompass a large group of more than 200 disorders that may lead to pulmonary fibrosis – an irreversible scarring of lung tissue that negatively impacts lung function.² When a progressive phenotype is present, a life-threatening condition can result that causes  difficulty breathing and a decrease in the amount of oxygen the lungs can supply to the body, measured through lung function decline.^3,4

"We believe nintedanib may help address an unmet medical need by providing a therapy for patients across a spectrum of ILDs with a progressive phenotype,” said Thomas Seck, M.D., senior vice president, Medicine and Regulatory Affairs, Boehringer Ingelheim Pharmaceuticals, Inc. “We are encouraged by this Breakthrough Therapy Designation and look forward to working closely with the agency to offer this therapy to patients for which there are no FDA-approved treatment options.”

The Breakthrough Therapy Designation process was established by the FDA to expedite the development and review of drugs for serious or life-threatening conditions where preliminary clinical evidence indicate that the therapy may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. In July 2014, the FDA granted nintedanib Breakthrough Therapy Designation for the treatment of people with idiopathic pulmonary fibrosis (IPF) and approved the drug for that use in October 2014.

About INBUILD
INBUILD is the first clinical trial in the field of ILDs to group patients based on the clinical behavior of their disease, rather than the primary clinical diagnosis.¹ The trial was a randomized, double-blind, placebo-controlled, parallel group trial conducted at 153 sites in 15 countries that evaluated the efficacy and safety tolerability of nintedanib (150 mg, 2 x daily) over 52 weeks in patients with progressive fibrosing ILD.¹

Results showed nintedanib slowed lung function decline by 57% across the overall study population, as assessed by the annual rate of decline in forced vital capacity (FVC) over 52 weeks in patients with fibrosing ILDs with a progressive phenotype.1 The most common adverse event was diarrhea, reported in 66.9% and 23.9% of patients treated with nintedanib and placebo, respectively, with a safety profile consistent to what has previously been seen in nintedanib.¹

About the disease
Patients with ILDs can develop a progressive phenotype that causes pulmonary fibrosis, leading to lung function decline, deterioration in quality of life and early mortality similar to IPF, the most frequent form of idiopathic interstitial pneumonias.³ The course of the disease and the symptoms are similar in progressive fibrosis ILDs regardless of the underlying disease.⁴ Chronic hypersensitivity pneumonitis, autoimmune ILDs such as rheumatoid arthritis-associated ILD, systemic sclerosis-associated ILD (SSc-ILD), mixed connective tissue disease-associated ILD, sarcoidosis and idiopathic forms of interstitial pneumonias, i.e. non-specific interstitial pneumonia, and unclassified idiopathic interstitial pneumonia are among these diseases.¹

About nintedanib
Nintedanib is already approved in more than 70 countries for the treatment of patients living with idiopathic pulmonary fibrosis (IPF) – a chronic and ultimately fatal disease characterised by a decline in lung function. In September 2019, nintedanib was approved in the U.S. as the first and only therapy to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated ILD. Submissions have been made to other major regulatory bodies across the globe. Boehringer Ingelheim is committed to improving the lives of people living with pulmonary fibrosis, in particular those affected by rare diseases with a high level of unmet need.

References

1 Flaherty K, et al. Nintedanib in Progressive Fibrosing Interstitial Lung Diseases N Eng J Med. Published 29 September, 2019. NEJM.org. DOI: 10.1056/NEJMoa1908681
2 British Lung Foundation. What is pulmonary fibrosis? Available at: https://www.blf.org.uk/support-for-you/pulmonary-fibrosis/what-is-pulmonary-fibrosis [Accessed September 2019].
3 Cottin V, Hirani NA, Hotchkin DL, et al. Presentation, diagnosis and clinical course of the spectrum of progressive-fibrosing interstitial lung diseases. Eur Respir Rev 2018;27(150):pii:180076.
4 Kolb M, Vašáková M. The natural history of progressive fibrosing interstitial lung diseases. Respiratory Research 2019:20:57.