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FG-3019 Granted Orphan Drug Designation for Treatment of Pulmonary Fibrosis

No FDA-approved therapies are available for the treatment of IPF, a progressive and fatal lung disease. FG-3019 was developed to inhibit the activity of CTGF, a matricellular protein that plays an important role in fibrosis. Research has established the critical role of CTGF as a final common pathway in chronic fibrotic diseases, in which persistent and excessive scarring leads to organ dysfunction and failure.

Orphan drug designation is generally granted to drugs or biologics intended for the treatment of rare diseases and disorders, i.e., those affecting fewer than 200,000 people in the U.S. This designation conveys special incentives to the sponsor, including tax credit for 50% of the cost of clinical trials, waiver of the prescription drug user fee, and seven years of U.S. market exclusivity for the drug or biologic upon FDA approval.

FG-3019 has been investigated in clinical studies involving more than 200 patients, including a phase I study of patients with IPF. FibroGen is currently conducting an open-label phase II study of FG-3019 in patients with IPF.

IPF is characterized by progressive scarring of the lungs that diminishes functional lung volume and hinders oxygen uptake. The cause of the disease is unknown. As scarring progresses, patients with IPF experience shortness of breath (dyspnea) and difficulty with performing routine functions, such as activities of daily living.

Approximately 40,000 cases of IPF are diagnosed annually in the U.S. and Canada. Approximately two-thirds of patients die within 5 years after diagnosis. Patients are often treated with corticosteroids and immunosuppressive agents; however, no drug treatments have been clinically proven to improve survival or quality of life.

For more information, visit the Fibogen Web site.

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