Pat Furlong, BSN, RN, on Progress With Research and Advocacy in Muscular Dystrophy
The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed how far the field has come since founding the organization/
“When I started PPMD 30 years ago, there was nothing, the field was totally empty. We didn't really didn't have much information on the patients. There was, in my view, therapeutic nihilism, this belief that there wasn't much to do. In fact, one doctor said to me, you know, maybe there will never be a treatment for Duchenne. I reacted very badly to that saying, yes, there will be and I'm certain of it.”
The field of Duchenne muscular dystrophy (DMD) has dominated its share of the newsfeed in the past year, with the breakthrough approval of delandistrogene moxeparvovec (Elevidys) gene therapy last summer, to the announcement of the global, pivotal, phase 3 EMBARK study (Study SRP-9001-301; NCT05096221) that supported Elevidys’ approval failing its primary end point in October 2023.1
Despite the negative news, clinically meaningful changes were observed in secondary end points and Sarepta Therapeutics has gone ahead and secured priority review for an efficacy supplement to its biologics license application (BLA) to remove requirements related to age and ambulatory-status (the approved indication is in children aged 4-5 while the trial included children up to age 7). The Prescription Drug User Fee Act date for the FDA’s decision on the supplement has been set for June 21, 2024.2
CGTLive spoke with Pat Furlong, BSN, RN, founding president and chief executive officer, Parent Project Muscular Dystrophy (PPMD), to learn more about her decades of experience in the field and how it has transformed in the time since PPMD’s founding to first gene therapy approval.
REFERENCES
1. Sarepta Therapeutics announces topline results from EMBARK, a global pivotal study of ELEVIDYS gene therapy for Duchenne muscular dystrophy. News release. Sarepta Therapeutics. October 30, 2023. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-topline-results-embark-global-0
2. Sarepta therapeutics announces U.S. FDA acceptance of an efficacy supplement to expand the Elevidysindication. News release. Sarepta Therapeutics, Inc. February 16, 2024. Accessed February 16, 2024.https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-us-fda-acceptance-efficacy?_ga=2.242068545.254033713.1708093077-1826905377.1708093076
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