Pamrevlumab

Unassigned

New Medicines

Non-ambulatory Duchenne muscular dystrophy

Information

New molecular entity
FibroGen
FibroGen

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials

Category

Anti-CTGF (connective tissue growth factor) human monoclonal antibody
Duchenne muscular dystrophy is a debilitating neuromuscular disease that affects approximately 1 in every 3,500 newborn boys. The fatal disease is caused by a genetic mutation leading to the absence or defect of dystrophin, a protein necessary for normal muscle function. The absence of dystrophin results in muscle weakness, muscle loss, fibrosis, and inflammation. Patients are often in a wheelchair by age 12 [1].
Non-ambulatory Duchenne muscular dystrophy
Intravenous infusion