There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community.
The clinical and research community have accelerated therapy development to our first-ever product reviews. Congress has ensured that regulators have the proper resources and authorities to review these products. Our families have spent years participating in the natural history studies and clinical trials needed to generate evidence of these products safety and efficacy profiles. And our Duchenne community has helped build a regulatory infrastructure complete with regulatory guidance and quantifiable patient preference data.
Please reach out to your Representative and urge them to sign on to the congressional letter being circulated by Representatives Fitzpatrick, Keating, and King on the Hill in support of the FDA’s continued use of the employment of the tools Congress included in the Food and Drug Administration Safety and Innovation Act (FDASIA) and the broad flexibility referenced in the Code of Federal Regulations Title 21.
This letter underscores FDASIA’s focus on accelerating the approval of drugs that treat unmet medical needs, prioritizing the patient perspective in evaluating new drugs and treatments, and providing regulators with flexibility when evaluating drugs for a life-threatening illness. Our community is heartened to know that our congressional champions remain committed to ensuring the FDA has the tools, authorities, and latitude necessary to review and clear safe, effective treatments for rare disease as quickly as possible.
Please urge your members to join by signing on and asking that the agency fully utilize these tools and authorities, especially where there is a favorable benefit-risk equation, to provide patients and physicians with new options to treat rare and deadly diseases like Duchenne.