Saracatinib, Potential Oral IPF Therapy, Granted Orphan Drug Status by FDA

Saracatinib (AZD0530), an investigational oral therapy under development by AstraZeneca, was granted Orphan Drug Designation as a potential treatment for idiopathic pulmonary fibrosis (IPF) by the U.S. Food and Drug Administration (FDA).

Saracatinib is a potent inhibitor of the SRC family of tyrosine kinases, a group of enzymes implicated in multiple cell signaling pathways that regulate important cellular functions, such as cell growth, differentiation, and survival.

The therapy was discovered by researchers at AstraZeneca, and has been in clinical development for different types of solid tumors. Saracatinib has been shown to inhibit tumor cell proliferation and tumor growth in animal models.

Preclinical tests done in human cells cultured in the lab and a mouse model of lung fibrosis showed that saracatinib inhibits two key events in lung fibrosis: it prevents collagen accumulation and the activation of fibroblasts — connective tissue cells key for wound healing and scarring (fibrosis).

The safety and tolerability of saracatinib has been evaluated in healthy volunteers. The therapy was given orally to volunteers as a single dose up to 1000 mg, and as multiple doses up to 250 mg per day during 14 days. Adverse events observed included anemia (low number of red blood cells or hemoglobin), nausea, anorexia, asthenia (physical weakness), fever, vomiting, diarrhea, and lung inflammation.

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AstraZeneca has or is currently conducting clinical trials evaluating the use of saracatinib as a treatment for alcoholism and Parkinson’s disease psychosis. In regard to IPF, Phase 2 studies addressing saracatinib’s efficacy and safety have not started yet.

FDA grants orphan drug status to medicines designed for the treatment, diagnosis or prevention of rare diseases or disorders that affect less than 200,000 people in the U.S. The status offers a series of incentives to companies developing those treatments, including tax credits for clinical testing.

“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, AstraZeneca’s executive vice president, R&D BioPharmaceuticals, said in a press release.

AstraZeneca also noted it has no plans to pursue evaluating saracatinib in additional preclinical or clinical oncology indications at this time.