Orphan drug status for saracatinib to treat IPFMay 8, 2019
The US FDA has granted Orphan Drug Designation (ODD) for saracatinib, a potential new medicine for the treatment of idiopathic pulmonary fibrosis (IPF).
Saracatinib is an inhibitor of src kinase which regulates broad cell functions including cell growth and cell differentiation. Saracatinib has completed phase I development.
IPF is a chronic, progressive, irreversible and usually fatal interstitial lung disease which affects approximately 100,000 people in the US. On average, patients who are diagnosed with IPF live between two and five years from diagnosis, given the limited medicines available to treat the disease.
IPF is characterised by thickening and scarring of the interstitial tissue in the lungs. The cause is thought to be due to an abnormal wound-healing process that results in excessive tissue build-up in the lung. Pre-clinical trials of saracatinib showed that it inhibits fibroblast activity and collagen deposition, which are key features of lung fibrosis.
Saracatinib is a small molecule, highly-potent and selective inhibitor of src tyrosine kinase. The potential new medicine was discovered by AstraZeneca and has previously been in clinical development in oncology. Phase II trials for saracatinib in IPF have not yet commenced.