Jeffrey S. Chamberlain, PhD, on Collaborating to Cure DMD
The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed the PDC group and collaborating with advocacy groups.
“There's a tremendous unmet need to treat the folks with Duchenne muscular dystrophy, and we feel that the data that's available is strongly supportive of approval. We want to bring together as many stakeholders as possible to evaluate the data, and then present that to the FDA to help them with their deliberations... This consortium has been together for quite a while and spent an enormous amount of time really critically evaluating [all the data on DMD]… We reached the conclusion that gene therapies can be highly effective, and that the production of a micro dystrophin protein can be quite beneficial and predictive of benefit... We’ve tried to outline the case, the rationale for everything we looked at, and make that publicly available for people to comment on and for the FDA totake a look at."
While the possibility of a gene therapy for treating Duchenne muscular dystrophy (DMD) is coming closer to reality, with Sarepta Therapeutics’ delandistrogenemoxeparvovec set for a PDUFA date of May 29, 2023, questions remain about approving gene therapies using accelerated approval pathways and surrogate endpoints. Such discussions even contributed to the FDA backtracking on an advisory committee meeting for Sarepta’s therapy, first saying a meeting was unnecessary in February 2023 and then announcing in March 2023 that a meeting would be held after all.1
A recent paper published in Human Gene Therapy by the Pathway Development Consortium (PDC) reviewed the use of mucrodystrophin as a surrogate endpoint in DMD trials and the suitability of the accelerated approval pathway.2 The authors, including first author Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, concluded that it is a valid endpoint that correlates with clinical benefits in preclinical mouse studies. CGTLive spoke with Chamberlain to learn more about the PDC, its goals, and its work with advocacy groups for DMD.
