FibroGen has just announced that their investigative therapy pamrevlumab, for Duchenne muscular dystrophy (DMD), has received Rare Pediatric Disease designation from the FDA. The treatment already was given Fast Track Designation. This therapy is currently being investigated in two different Phase 3 investigations for DMD.
Duchenne muscular dystrophy is a rare condition that affects predominately males. It is a neuromuscular condition caused by either a defect or the absence of a protein called dystrophin. This is a result of a genetic mutation.
Without dystrophin (or with a defective dystrophin protein) muscles cannot function normally. It leads to fibrosis, muscle loss, weakness, as well as inflammation.
Currently, most patients start exclusively using a wheelchair by the age of 12. Many individuals also face other medical issues due to cardiac and respiratory muscle weakness.
This investigative therapy is an antibody which works to stop the activity of CTGF- the connective tissue growth factor. CTGF is a critical mediator within proliferative and fibrotic conditions.
For details on all of these trials, please reference clinicaltrials.gov.
You can read more about FibroGen’s work and this new investigative therapy for DMD here.