The U.S. Food and Drug Administration (FDA) has accepted for filing Boehringer Ingelheim's New Drug Application (NDA) for nintedanib, an investigational compound intended for the treatment of idiopathic pulmonary fibrosis (IPF). The FDA has also granted the application Priority Review designation, expediting the review process for this potentially life-saving therapy.
IPF is a rare, progressive, and fatal lung disease characterized by scarring of the lung tissue, leading to shortness of breath and reduced lung function. Currently, there are no FDA-approved treatments available to halt or reverse the progression of IPF, representing a significant unmet medical need.
The NDA submission is based on data from two global Phase III studies, INPULSIS-1 and INPULSIS-2, which evaluated the efficacy and safety of nintedanib in patients with IPF. These trials assessed the impact of nintedanib on lung function decline, measured by forced vital capacity (FVC), a key indicator of disease progression.
Nintedanib is a small molecule tyrosine kinase inhibitor (TKI) that targets several growth factor receptors implicated in the pathogenesis of pulmonary fibrosis. These include vascular endothelial growth factor receptor (VEGFR), fibroblast growth factor receptor (FGFR), and platelet-derived growth factor receptor (PDGFR). By blocking these receptors, nintedanib aims to reduce fibroblast proliferation and collagen deposition, thereby slowing the progression of IPF.
In June 2011, nintedanib received orphan drug designation in the US, a status granted to therapies intended for rare diseases. The Priority Review designation by the FDA underscores the urgent need for effective treatments for IPF and the potential of nintedanib to address this critical gap in care.
