New Drug for Duchenne Muscular Dystrophy (DMD) Shows Promise

FibroGen’s monoclonal antibody pamrevlumab provided positive results in a small, early study. 

FibroGen is developing its drug pamrevlumab for the treatment of non-ambulatory DMD patients and people with idiopathic pulmonary fibrosis (IPF) and locally advanced pancreatic cancer. Initial results from a small single-arm study of 21 DMD patients who cannot walk were recently announced.

Data collected for 52 weeks were analyzed for this first report. The study is continuing for up to 156 weeks. Treatment with pamrevlumab, which is a monoclonal antibody to connective tissue growth factor (CTGF), resulted in a slowing of the rate of decline of pulmonary function, a common cause of morbidity and mortality in non-ambulatory patients, improved cardiac function, a reduction in cardiac fibrosis, improved grip strength and reduced loss of upper arm function.

FibroGen will need to conduct a blinded, placebo-controlled, randomized pivotal trial in DMD patients who cannot walk to confirm these early results. The company is currently starting phase III trials in IPF and pancreatic cancer. It hopes to launch the drug in 2022.

David Alvaro, Ph.D.

David is Scientific Editorial Director for That’s Nice and the Pharma’s Almanac content enterprise, responsible for directing and generating industry, scientific and research-based content, including client-owned strategic content. Before joining That’s Nice, David served as a scientific editor for the multidisciplinary scientific journal Annals of the New York Academy of Sciences. He received a B.A. in Biology from New York University and a Ph.D. in Genetics and Development from Columbia University.