The senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen discussed why turning Duchenne muscular dystrophy into a chronic disease rather than a fatal one, is a top priority.
"I’m looking forward for the patient to live with Duchenne, but not die from Duchenne. That will be the greatest accomplishment.”
FibroGen’s pamrevlumab enters a phase 3 trial as another drug hoping to become FDA-approved for the treatment of Duchenne muscular dystrophy (DMD), adding to the growing number of treatments available for patients. The drug will be assessed in both ambulatory and non-ambulatory populations, addressing 2 major types of patients with DMD that are at different stages of disease progression.
As the momentum within the DMD research community continues to surge, there still remains a great unmet need according to Elias Kouchakji, MD. Kouchakji, who is the senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen, feels as though transforming the fatal disease to a chronic one is among the most pressing needs at the moment.
Kouchakji compared the goals of DMD research to that of HIV, where instead of dying from it, patients are dying with it. In an interview with NeurologyLive, Kouchakji explained what he feels is the greatest unmet need, and how close the DMD research community is to achieving that goal.