FibroGen Gains Orphan Status for Duchenne Therapy

FDA has granted FibroGen an orphan drug designation for the company’s anti-connective tissue growth factor (CTGF) antibody, pamrevlumab, for treating patients with Duchenne muscular dystrophy (DMD). The designation is based on data from a Phase 2 study in 21 non-ambulatory DMD patients who have completed the first 52 weeks of treatment. “We are evaluating a number of clinical parameters in this study, including lung function, cardiac function, and upper extremity muscle function, and tissue fibrosis,” the company says.

Pamrevlumab is a first-in-class antibody developed by FibroGen to inhibit the activity of CTGF, which the company describes as a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Fibrogen says it is also advancing toward Phase 3 clinical development of pamrevlumab for treating idiopathic pulmonary fibrosis and pancreatic cancer.