Paul Melmeyer on Addressing Gene Therapy Regulatory Bottlenecks


The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval.

“Over the course of the last several years, we've been working on trying to strengthen the FDA’s capacity, expertise, and ability to quickly review gene therapies that come in front of them. The Office of Tissues and Advanced Therapies in the Center for Biologics Evaluation and Research (CBER), who is responsible for gene therapy reviews, has been underfunded and understaffed for years at this point. We are very happy that due to our and many other organizations’ advocacy, we are able to successfully enact provisions within the FDA User Fee Reauthorization... That would greatly expand this office, that would actually elevate this office to be The Office of Tissue Products within CBRE..."

Many investigational gene therapies are currently in development that have the potential to address unmet needs for patients with neuromuscular diseases, including muscular dystrophy. However, bottlenecks, understaffing, and inefficiencies in regulatory processes can hold back the review and approval of these therapies.

In an interview with CGTLive, Paul Melmeyer, vice president, Public Policy & Advocacy, Muscular Dystrophy Association (MDA), who recently spoke at the 2022 NORD Breakthrough Summit in Washington, DC, discussed the organization’s work with regulatory agencies to help address these issues. He mentioned the specific steps MDA has taken with the FDA regarding the Office of Tissues and Advanced Therapies and provisions pertaining to a patient-focused drug development meeting on gene therapies. Melmeyer also highlighted several additional regulatory and legislative goals he sees as the highest priorities for the patient community. He concluded by mentioning that neurologists and other allied medical professionals can help support the organization’s goals for December.

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