CureDuchenne Submits Comments on FDA’s Draft Guidance for Industry on Platform Technology Designation Program

CureDuchenne welcomes the recent initiative the Food and Drug Administration (FDA) has taken to improve efficiencies in drug development, manufacturing, and the review process for new drug applications that incorporate designated platform technologies. This initiative is designed to help streamline the drug development and approval process, allowing drug companies to leverage prior knowledge from their previously submitted applications. 

The FDA has recently requested comments and suggestions to help establish the final Platform Technology Designation rules.

As the leading patient advocacy organization, CureDuchenne is committed to keeping the community updated on the progress of this initiative as we believe that this could positively impact a number of companies developing exon-skipping therapies by lowering the barrier to develop drugs for very rare mutations.

Read the FDA Draft Guidance

Comments Submitted by CureDuchenne to the FDA (Submitted 8/27/24):

CureDuchenne appreciates the opportunity to provide comments to the FDA regarding the draft guidance document “Platform Technology Designation Program; Guidance for Industry” which appeared in the Federal Register on May 28, 2024.

Duchenne Muscular Dystrophy (DMD) is the most common of the childhood muscular dystrophies, affecting approximately 1 in 5,000 newborn males. All cases of DMD are caused by mutations in the DMD gene, which result in a lack of production of the dystrophin protein in muscle cells. Without dystrophin, muscles are prone to injury and dysfunction, and most individuals with DMD will lose the ability to walk in their early teenage years. Eventual weakness of the respiratory muscles and/or cardiomyopathy, typically in the third decade of life, is the most common cause of death. While there are several FDA-approved therapies that slow this progression of disease, there are no treatments that are curative.

The DMD gene is the largest gene in the human genome, and there are thousands of reported mutations that result in DMD. One strategy to restore the dystrophin RNA reading frame utilizes exon skipping. Exon skipping aims to bypass, or skip over, a particular exon in the dystrophin RNA, to counteract the problems caused by a disease-causing mutation. Exon skipping aims to maximize a process that occurs at low levels naturally in some DMD individuals, and is often associated with a milder progression of disease. Since exon skipping is a mutation-specific approach, it won’t work for every individual with DMD. Theoretically, up to almost 80% of people with DMD have genotypes that are amenable to exon skipping. However, each of these subpopulations are relatively small. The four FDA-approved exon skipping drugs, which skip exons 51, 53 or 45, collectively are appropriate for only 29% of individuals with DMD. Exon skipping programs for other exons