Earlier today, Saol Therapeutics shared the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding the company’s application for dichloroacetateInvestigational drug for treatment of mitochondrial disease. A small inorganic molecule that increases the rate of oxidative phosphorylation, the main pathway by which the energy in food is converted (DCA) to treat Pyruvate Dehydrogenase Complex Deficiency (PDCD). In this CRL, the FDA declined to approve DCA at this time and requested that Saol initiate a new clinical trial to address remaining questions. Attached you will find a community statement from Saol.
We are deeply disappointed by this outcome and mindful of what it means for families awaiting options. Our collective organizations and Saol remain committed to advocating for timely, patient-centered next steps, engaging with regulators, and keeping you informed as more details emerge.
In response to this decision, we have joined with fellow PDCD and mitochondrialRelated to the mitochondria. disease-related patient advocacy groups to issue the below statement and call to action.
On behalf of the mitochondrial disease community, we, the United Mitochondrial Disease Foundation, MitoAction, Cure Mito Foundation, Hope for PDCD, and the Elizabeth Watt PDCD Research Fund, collectively express our deep disappointment with the FDA’s recent decision not to approve dichloroacetate (DCA) for the treatment of the mitochondrial disease Pyruvate Dehydrogenase Complex Deficiency (PDCD).
While we fully support rigorous scientific standards, regulatory flexibility is essential for rare disease populations like PDCD, where delays in access to potentially life-saving therapies can lead to irreversible damage – or even death. Make no mistake, that is the risk when it comes to DCA not being available for PDCD patients.
PDCD is a genetic disorder of carbohydrate metabolism and the most common cause of congenital lactic acidosis. Families in the DCA trial report significant quality of life improvements.
There are patients in our community who have been on some form of this therapy for nearly 30 years. This well-understood drug with a strong safety profile is supported by both patients and clinicians. The approval of DCA would have addressed a profound unmet medical need for affected PDCD patients in the US. Instead, another generation of PDCD patients face an uncertain future.
The FDA has statutory authority, and a moral and ethical obligation, to apply flexibility when evaluating therapies for life-threatening rare diseases like PDCD. When this authority is not exercised, it raises serious questions about whether the rare disease drug approval pathway is truly serving the patients it was intended to help.
We are asking the FDA to continue to work with Saol Therapeutics to find an expedited path forward for DCA. There is simply too much on the line for any other option. As the unified voice of the mitochondrial disease community, we will not stop advocating until PDCD patients gain access to the therapies they need and deserve.
Our groups will be scheduling a call to go over some suggested talking points and the most effective way to tell the story of PDCD and DCA shortly. Until then, we encourage you to reach out to your members of Congress to help them better understand PDCD and the importance of regulatory flexibility for therapies like DCA. Click HERE for a form letter that will be sent straight to your representative.
Saol remains committed to this community and plans to host a community town hall on Thursday, Sept. 18 at 3pm ET. Watch for an invite in the coming days. We encourage you to attend and submit whatever questions you may have.
Sincerely,
United Mitochondrial Disease Foundation
MitoAction
Cure Mito Foundation
Hope for PDCD
Elizabeth Watt PDCD Research Fund
Click HERE to read the full Community Letter from Saol Therapeutics
