The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for saracatinib, a potential new medicine for the treatment of idiopathic pulmonary fibrosis (IPF), a type of lung disease that results in scarring (fibrosis) of the lungs. 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 disease1 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. The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.
“Idiopathic pulmonary fibrosis has a significant impact on patients’ lives and new therapies are urgently needed. IPF is a recent addition to our respiratory research strategy and we are interested to see whether saracatinib could be a useful approach for the treatment of this intractable disease,” Mene Pangalos, Executive Vice President, R&D BioPharmaceuticals, said.
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IPF is characterized by thickening and scarring of the connective (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.
IPF causes shortness of breath and progressive damage of the lung, resulting in life-threatening complications such as respiratory failure. IPF progression varies greatly between patients but over time, most experience increasing respiratory symptoms, increased scarring of the lungs and a gradual decline in lung function. ‘Idiopathic’ refers to the unknown cause of disease, however there is proof of genetic predisposition in some patients.
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.