AstraZeneca's Saracatinib Receives FDA Orphan Drug Designation for Idiopathic Pulmonary Fibrosis

VCBeat (WeChat ID: vcbeat) has learned that on March 18, 2019, the biopharmaceutical company AstraZeneca announced that its drug Saracatinib had received FDA Orphan Drug designation for the treatment of idiopathic pulmonary fibrosis.

It is understood that the FDA defines rare diseases, also known as orphan diseases, as those affecting fewer than 200,000 people in the United States, and grants orphan drug designation to breakthrough therapies intended to treat these conditions. Due to the small patient population, limited market demand, and high research and development costs, few pharmaceutical companies focus on developing treatments for rare diseases. Therefore, the FDA has established a series of incentives; companies developing orphan drugs are eligible for preferential policies, including tax credits for clinical trial expenses, waivers of FDA user fees, FDA assistance with clinical trials, and seven years of market exclusivity upon drug approval.

Saracatinib is a small-molecule Src tyrosine kinase inhibitor that modulates cellular processes such as cell growth and differentiation. Phase I clinical trials have been completed, with results demonstrating that Saracatinib inhibits fibroblast activity and collagen deposition, thereby preventing the development of pulmonary fibrosis. AstraZeneca is now poised to initiate Phase II clinical trials.

Idiopathic pulmonary fibrosis is an irreversible and fatal interstitial lung disease that affects approximately 100,000 people in the United States. The term "idiopathic" indicates that the etiology is unknown, although evidence suggests a genetic predisposition in certain patients. As a respiratory disorder, it presents with symptoms such as dry cough and dyspnea, ultimately leading to death from respiratory failure. Corticosteroids, immunosuppressants, and cytotoxic agents are the primary pharmacological treatments; however, existing medications can only slow disease progression and are associated with toxic side effects. Due to the limited therapeutic options available for idiopathic pulmonary fibrosis, patients have a survival period of only 2 to 5 years following diagnosis.

Mene Pangalos, Executive Vice President at AstraZeneca, stated: “Idiopathic pulmonary fibrosis significantly impairs patients’ quality of life, and there is an urgent need for new treatment options. This disease is one of our company’s key research focuses, and we are confident in developing saracatinib as an effective therapy for this refractory condition.”

AstraZeneca, founded in 1913 and headquartered in London, United Kingdom, is a biopharmaceutical company dedicated to providing innovative prescription medicines for patients across multiple therapeutic areas, including gastrointestinal, cardiovascular, oncology, central nervous system, anesthesia, respiratory, and anti-infective diseases. Many of its products, such as Tagrisso, Brilinta, and Pulmicort Respules, hold leading positions globally, with sales covering more than 100 countries and regions worldwide. AstraZeneca entered the Chinese market in 1993.

(Compiled by Jiao Yanli)