The FDA has awarded orphan drug status to AstraZeneca’s AZD0530 (Saracatinib), a potential new drug for the treatment of idiopathic pulmonary fibrosis (IPF).
IPF, a chronic, progressive, irreversible, and typically fatal interstitial lung disease, affects approximately 100,000 people in the U.S. Patients with IPF generally live between two and five years from diagnosis, as there are few medicines available for treating the disease.
Pre-clinical trials of AZD0530 showed that it inhibits fibroblast activity and collagen deposition, which are key features of lung fibrosis.
AZD0530 is a small molecule, highly potent, selective inhibitor of src tyrosine kinase, which regulates broad cell functions including cell growth and cell differentiation. Phase 2 trials for AZD0530 in IPF have not yet commenced.
Source: AstraZeneca, March 18, 2019