FOLSOM, Calif., Dec. 11, 2025 (GLOBE NEWSWIRE) -- The Hope for PDCD Foundation, a parent-led patient advocacy group dedicated to advancing research and treatments for the rare mitochondrial disease Pyruvate Dehydrogenase Complex Deficiency (PDCD), today announced that it has delivered a petition to the U.S. Food and Drug Administration urging approval of the first targeted potential treatment for PDCD. The wider mitochondrial disease advocacy community, including the United Mitochondrial Disease Foundation, MitoAction, the Cure Mito Foundation, and the Elizabeth Watt PDCD Research Fund, coalesced to support the petition, and it rapidly garnered more than 17,000 signatures in less than two months.

The FDA declined to approve a targeted treatment for PDCD – sodium dichloroacetate (DCA) – in September 2025 despite encouraging clinical evidence and the voices of PDCD families reinforcing the marked improvement their children experienced while being treated with DCA.

“While being treated with DCA, children with PDCD, including my daughter Violet, have achieved gains in quality of life, stability, and developmental milestones that many parents in our community never dreamed possible,” said Frances Pimentel, founder and chief executive officer of the Hope for PDCD Foundation and PDCD mom. “We ask the FDA, publicly and directly, to listen to PDCD families, meet our children, and hear our stories. We want a seat at the table to help shape a path forward and ensure our community has access to potentially life-saving treatments.”

More than 80 leading researchers and clinicians in the mitochondrial disease space signed a joint letter of support urging FDA to pursue an expedited, flexible regulatory path forward for DCA as the first and currently only potential treatment for PDCD. In addition, five mitochondrial disease patient advocacy groups issued a statement expressing deep disappointment in the FDA’s decision.

“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,” said the advocacy groups in the joint statement.

Children with PDCD often die in infancy. Many of those who survive face debilitating neurological, developmental, and physical challenges, including severe energy depletion, intellectual disability, limited mobility, inability to communicate, developmental delay, and irreversible brain damage. PDCD is a devastating, life-threatening, progressive condition. The only way for PDCD kids to survive is to adhere to a strict ketogenic diet that is harsh on the body and associated with serious complications. Even on a rigid keto diet, PDCD kids continue to experience recurring hospitalizations, a need for mobility devices, and medical support for many other daily activities.

Parent Testimonials

As part of the petition, parents of children with PDCD shared testimonials about the improvements they observed after their children began treatment with DCA:

“[My daughter] Harlow entered the [DCA] trial…and we saw changes no one could deny. She had more energy. Her mind was sharper. Her quality of life improved. DCA gave her what the ketogenic diet alone could not: stability, relief, and a glimpse of possibility,” wrote Kim Higbee, Hope for PDCD Foundation board member, in an op-ed. 

“Within a couple weeks of starting DCA, we began to see changes [in our daughter, Talia]. We saw a new joy and lightness in her, as if she could forget, even briefly, some of the pain and exhaustion she was facing, and just be a kid. She took her first steps at 3 years old, just 4 months after starting DCA. Within one year, she transitioned from a feeding tube to eating and drinking completely by mouth,” said Meagan DeRaps, creator of the ApproveDCA.com website. 

“We want the FDA to sit down with us, sit down with the PDCD patient community and hear our stories… If [DCA is] beneficial even a tiny bit, that is going to improve the quality of [my daughter Piper’s] life and the length of her life. So we’re willing to take that risk – especially if there really isn’t a risk if the FDA has said that it’s safe,” said Layna O’Connor, Hope for PDCD Foundation board member, in an interview with West Michigan Channel 13 News.

DCA Clinical Data

In a webinar presented to the PDCD community, results of the national Phase 3 clinical trial of DCA in PDCD patients were summarized. In the study, PDCD patients demonstrated functional improvement in those with more serious disease, and a reduction in lactate, a known biomarker of the disease. When compared to historic cohorts, treatment with DCA has substantially reduced mortality in patients with PDCD. DCA is also a safe, well-understood intervention.

Saol Therapeutics, the small, privately held company behind the DCA clinical program, stated in a recent press release that they shared new data with FDA as part of the Type A meeting process where a pathway to approval will be discussed. Analyses from the open-label extension study showed evidence that longer DCA treatment has resulted in sustained developmental improvement in children with PDCD. The company also submitted additional data on survival benefits, mechanistic support, and safety.

About the Hope for PDCD Foundation

Founded in 2022, the Hope for PDCD Foundation is dedicated to advancing research and treatments for the rare mitochondrial disease Pyruvate Dehydrogenase Complex Deficiency (PDCD). The Foundation is entirely run by volunteers, the majority of whom are parents of children with PDCD. Learn more at hopeforpdcd.org or connect with us on Facebook, Instagram, X, and LinkedIn.

Media Contact
Jessi Rennekamp, Astrior Communications
Email: jessi@astriorcomms.com