FDA Announces first Platform Technology Designation: Implications for CMD’s Path to Clinical Trials

Recently, there’s been a movement in the pharma space to create "platform technology" companies - systems that can be used to treat a type of mutation (e.g., splice site, nonsense, etc.) within a class of diseases, rather than a specific disease or gene.

The Concept

A platform technology enables the reuse of previously tested and validated components of a therapeutic product, eliminating the need for repeated human safety testing. Developers must only demonstrate that any newly introduced module, when combined with approved components, has minimal differences from an existing, authorized drug or biologic.

Establishing Similarity

To ensure regulatory approval, developers must provide evidence that the new module closely resembles existing components in terms of:

• Structure (e.g., molecular composition and chemical properties)

• Mechanism of Action (how the drug interacts with biological systems)

• Biological Effect (expected therapeutic outcomes)

• Manufacturing Process (production methods ensuring quality and safety)

• Preliminary Evidence

• Applicants should leverage relevant data to support the similarity between the new and existing modules. This could include:

• Structurally similar drug substances (e.g., comparable nucleic acid sequences with similar backbone chemistry and targeting mechanisms)

• Minimal differences in drug formulation (both qualitatively and quantitatively)

• Nearly identical manufacturing and purification processes

Example: Gene Therapy

For a gene therapy, the following core modules may be used (we’ll use the COL6A1 gene as an example):

1. Viral vector (e.g., AAV9 for delivering the gene)

2. Safe dosage range (approved levels for administration)

3. Immunosuppression protocol (to prepare the patient’s immune system)

4. Therapeutic gene construct (e.g., COL6A1 gene driven by the muscle-FAP specific promoter )

5. Manufacturing process (ensuring consistency and quality)

If say, a new gene therapy is developed for a related gene, COL6A2, modules 1-3 and 5 remain unchanged, and only module 4 (the gene construct) is replaced with the COL6A2 gene. The only regulatory requirement will be to demonstrate that COL6A2 is sufficiently similar to the therapy already approved for COL6A1, rather than being required to re-validate the entire therapy from scratch.

First Platform Technology Designation

Just this month, the FDA granted its first "Platform Technology Designation" to Sarepta Therapeutics, a company developing treatments for Duchenne and several LGMDs. This first designation will now carve a path for other companies developing similar treatment approaches.

“This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,” said Louise Rodino-Klapac, PhD, chief scientific officer and head of research & development, Sarepta.

The FDA’s new platform technology designation program is a critically important tool to support the development of treatments for rare conditions like the congenital muscular dystrophies, for which low prevalence has historically been a near-insurmountable hurdle to garnering pharmaceutical interest. This new technology aims to allow for the streamlining of drug development, manufacturing, and the review process for future FDA applications - all great news for rare and ultra rare conditions like ours.

This FDA approval is the kind of momentum Cure CMD has been working toward for years. But to take advantage of it, we need the resources to keep pushing forward - connecting with researchers and funding their most promising work, providing valuable insights to industry, and making sure no subtype gets left behind. If you've ever thought about making a significant gift to Cure CMD, now is the time. Your support could help turn this breakthrough into real hope for our CMD community.

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Update July 19, 2025: The FDA revoked Sarepta's platform technology designation in light of recent safety concerns around current clinical trials. The designation still remains an option to pursue with the FDA for pharmaceutical companies working on similar platforms.