Ask Congress to Encourage "Regulatory Flexibility" for Rare Disease Therapies like DCA

We encourage you to use this form to help your members of Congress better understand PDCD and the importance of regulatory flexibility for therapies like DCA.

Simply input your address in the form to the right, click "next" and then "send email" to get the below letter to your Congressional offices.

On September 4, 2025 Saol Therapeutics shared a press release stating that the U.S. Food and Drug Administration (FDA) will not approve Saol Therapeutic’s New Drug Application for SL1009 (DCA) for Pyruvate Dehydrogenase Complex Deficiency (PDCD) in its current form, outlining specific observations that must be addressed before potential approval can be reconsidered. 

While we fully support rigorous scientific standards, regulatory flexibility is essential in the rare disease community and we are asking that you, our representative, please reach out to senior officials at the Department of Health and Human Services (HHS) to discuss the value and importance of regulatory flexibility in the rare disease space!

PDCD is a rare, life-threatening, genetic, mitochondrial disorder of carbohydrate oxidation that affects the nervous system and skeletal muscle and leads to decreased energy production. DCA is a targeted therapy that increases energy production by the mitochondria. Currently, no drug therapies exist for PDCD.

The FDA’s own rare disease guidance affirms that it may accept greater uncertainty and encourages the use of natural history, real-world data, and innovative trial designs.  Ultimately, the FDA created regulatory flexibility for the rare disease communities, like ours, because it is so important to bring meaningful treatments to “rare” communities.

We are asking you that you please encourage regulatory flexibility for rare disease. Under the current guidance that the FDA is providing Saol Therapeutics, it would take “several years and millions of dollars for an approval,” according to Saol’s press release. How is this approach utilizing regulatory flexibility?

We appreciate your willingness to discuss the importance of regulatory flexibility in the rare disease space with HHS Secretary Robert F Kennedy, Jr. and senior officials.