Pamrevlumab Gets Orphan Drug Designation for Duchenne Muscular Dystrophy

The Food and Drug Administration (FDA) has granted Orphan Drug designation to pamrevlumab (FibroGen), for the treatment of patients with Duchenne muscular dystrophy (DMD).

Pamrevlumab is a first-in-class fully human antibody designed to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders. According to the company, an ongoing phase 2 study in 21 non-ambulatory DMD patients has passed the 52-week treatment mark. “We are evaluating a number of clinical parameters in this study, including lung function, cardiac function, and upper extremity muscle function, and tissue fibrosis,” stated Elias Kouchakji, MD, Senior Vice President, Clinical Development and Drug Safety.

The FDA’s Orphan Drug designation is granted to treatments for rare diseases that affect ≤200,000 individuals. Additionally pamrevlumab has received Fast Track designation for the treatment of idiopathic pulmonary fibrosis and locally advanced unresectable pancreatic cancer. The company has conducted phase 2 trials in these populations.

“We are pleased to have received Orphan Drug designation from the FDA for pamrevlumab in the treatment of DMD. There is high unmet medical need for patients suffering from this debilitating disease needing a new treatment option,” said Dr Kouchakji.

For more information visit fibrogen.com.