Elias Kouchakji, MD: Importance of Non-Ambulatory Patients in DMD Trials

October 16, 2020

The senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen discussed why the representation of non-ambulatory patient populations is necessary in DMD trials.

"Putting an estimate of survival, or how long they’re going to be living, is to also take a look at how long you’re going to maintain their living into more functional.”

An important phase in Duchenne muscular dystrophy (DMD) is the loss of ambulation, which can usually occur between the ages of 7 and 13 years, leaving patients in a wheelchair and damaging their quality of life. During that time, their lungs may weaken, which can lead to difficulties in breathing and an increased risk of chest infections. For this reason, DMD gene therapy trials over the years have often been tailored towards ambulatory patients.

Elias Kouchakji, MD, senior vice president of clinical development, drug safety, and pharmacovigilance, FibroGen feels as though these patient populations have been underrepresented but are slowly making their way into more clinical trials. Pamrevlumab, Fibrogen’s investigational DMD agent, is expected to be assessed in a phase 3 setting that will include both ambulatory and non-ambulatory patients.

Like many others, Kouchakji has stressed that a more rigorous examination of this patient population will support the general hypothesis with neurological disorders in that, earlier treatment can improve quality of life features further down the clinical disease progression. In this interview with NeurologyLive, Kouchakji takes a closer look at this population, and why he feels it holds a sacred place within the DMD community and research settings.