Boehringer Ingelheim recently announced European Union (EU) approval of nintedanib (brand name OFEV®) for idiopathic pulmonary fibrosis (IPF) treatment. The committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion, which means that the treatment can now be marketed, sold, and used in humans in the EU. The United States Food and Drug Administration approved nintedanib in October.
Idiopathic pulmonary fibrosis is a chronic, fatal disease that causes lung scarring (fibrosis) and loss of lung function. Few treatment options exist for this condition, and current interventions include oxygen, lung rehabilitation, and lung transplant. Worldwide, IPF is estimated to affect 14–43 people in every 100,000.
The EU approval was based on the Phase III INPULSIS® trial results, published in the New England Journal of Medicine in May. These studies showed that nintedanib significantly slowed IPF disease progression.
Professor Klaus Dugi, Chief Medical Officer, Boehringer Ingelheim stated “Boehringer Ingelheim welcomes the decision by the CHMP. There has been a high unmet need for effective treatments that can slow disease progression in IPF. We look forward to making nintedanib available soon to patients with IPF in the EU.”
The two INPULSIS® trials included 1,066 patients in 24 countries. Oral nintedanib was taken twice daily for 52 weeks. The main measurement that the researchers used to tell whether the drug was effective was “forced vital capacity” an assessment of lung function, during which the patient is asked to exhale forcibly and the researcher measures the volume of air that comes out. The trials also took into account quality of life related to healthcare concerns, and how long it took until “first acute exacerbation” — which is a sudden worsening of lung and breathing symptoms.
In these Phase III clinical trials, researchers found that nintedanib slowed the worsening of lung function problems caused by IPF by 50% compared to patients taking a sugar pill, and nintedanib reduced the risk of acute exacerbations by 68%. One study but not the other showed that quality of life improved in the group taking nintedanib compared to the group taking a sugar pill.
Side effects caused by nintedanib were mostly mild or moderate gastrointestinal problems. These were generally manageable and in almost all cases did not cause patients to stop taking the medication.
INPULSIS® study investigator Professor Luca Richeldi, Professor of Respiratory Medicine, Chair of Interstitial Lung Disease at the University of Southampton, United Kingdom remarked “This decision is very encouraging as patients with IPF currently have very limited treatment options. For the first time we have a drug that has consistently met the primary endpoint in two large Phase III trials, confirming the results of the Phase II trial.”