Leading advocacy groups and more than 80 scientific and medical leaders call on FDA to act urgently on DCA approval for PDCD, a rare and life-threatening mitochondrial disease
WASHINGTON, Oct. 16, 2025 /PRNewswire-PRWeb/ -- In response to the recent Complete Response Letter (CRL) issued by the U.S. Food and Drug Administration (FDA) regarding Saol Therapeutics' application for dichloroacetate (DCA) to treat Pyruvate Dehydrogenase Complex Deficiency (PDCD), leading patient advocacy organizations, including the United Mitochondrial Disease Foundation, MitoAction, Cure Mito Foundation, Hope for PDCD, and the Elizabeth Watt PDCD Research Fund, today announced more than 80 of the leading researchers and clinicians in the mitochondrial disease space have signed a joint letter of support urging the FDA to pursue an expedited, flexible regulatory path forward.
PDCD is a rare genetic disorder of carbohydrate metabolism and the most common cause of congenital lactic acidosis. It can lead to developmental delays, neurologic dysfunction, and early death. DCA, a therapy with decades of patient experience and a favorable safety profile, has been used in research and clinical practice for nearly 50 years, with families reporting significant improvements in quality of life.
"PDCD is a very serious pediatric neurological disease, where children can have intractable seizures, can lose developmental milestones, suffer irreversible neurologic injury, and, in many cases, face death in early childhood," said Dr. Rebecca Ganetzky MD, Pediatrician, Biochemical Geneticist, and co-author of the physician letter. "For decades, dichloroacetate (DCA) has shown the ability to reduce lactic acidosis and stabilize or improve function in PDCD patients. Every day without access to DCA is a day when preventable harm may be occurring. The FDA has the authority to apply flexibility for ultra-rare diseases like PDCD, where the clinical benefit is clear and the consequences of delay are devastating and it is critical to work together to facilitate access to therapies for ultra-rare diseases."
Dr. Peter Stacpoole PhD, MD, Endocrinologist, long-time PDCD researcher, and co-author of the clinician letter, added: "DCA is one of the most studied compounds in the history of PDCD and has demonstrated meaningful benefit for patients. The global medical community's support of this therapy underscores the urgent need to bring it to families in the U.S. who are out of options."
While acknowledging the FDA's mandate for rigorous review, the organizations emphasized that statutory provisions exist to allow flexibility in rare, life-threatening diseases. The CRL requested that Saol Therapeutics initiate an additional randomized controlled clinical trial, a requirement that is both cost- and time-prohibitive, before approval can be granted, raising concerns that another generation of patients will go without access to a potentially life-saving therapy.
"We are united in saying that the status quo is not acceptable," the groups collectively expressed. "PDCD families deserve timely access to treatments. The FDA must act with urgency, flexibility, and compassion to prevent avoidable suffering."
The advocacy organizations pledged to continue collectively mobilizing the PDCD community, engaging regulators, and elevating the voices of both families and clinicians until an appropriate and expedited path forward is secured.
About the United Mitochondrial Disease Foundation
For nearly 30 years, the United Mitochondrial Disease Foundation (UMDF) has built a global network of patients, researchers, clinicians, institutions, and industry partners dedicated to fighting mitochondrial disease. Together with the mito community, UMDF utilizes a four-pronged strategy of supporting and connecting affected families, helping train and educate the medical community, working with policymakers for mito-friendly legislation, and helping find research dollars to unlock the science needed for treatments and eventual cures. Learn more at umdf.org.
About MitoAction
MitoAction is a national nonprofit dedicated to improving the lives of children, adults, and families affected by mitochondrial disease. We support patients and caregivers through a wide range of programs, educate the broader community about the impact of these rare disorders, and advocate for policies that advance access and awareness. From wish trips and family support services to driving forward research and therapy development, MitoAction is committed to empowering the mitochondrial disease community with hope, resources, and a voice for change. Learn more at mitoaction.org.
About Cure Mito Foundation
Cure Mito Foundation is a parent-led 501(c)(3) nonprofit uniting the global Leigh syndrome community to accelerate patient-centered research, treatments, and cures. The foundation funds groundbreaking research and empowers families while advancing collaboration through the Leigh Syndrome Global Patient Registry, the annual Empower & Inspire Leigh Syndrome Symposium, practical tools for researchers and healthcare providers, and accessible support and educational resources. Cure Mito leads Leigh Syndrome Awareness Day each September 17 to honor those affected and raise global awareness. Cure Mito Foundation is proud to be part of the Chan Zuckerberg Initiative (CZI) Rare As One Network, supporting collaboration and innovation across the rare disease community. Learn more at curemito.org.
About Hope for PDCD
Hope for PDCD was founded in 2022 with an urgent mission: to cure Pyruvate Dehydrogenase Complex Deficiency. Hope for PDCD's core values are: 1) to empower PDCD patients and families, 2) to promote solidarity among the PDCD community and 3) to build a better future for PDCD. All financial gifts are invested wisely and 100% of every dollar donated goes to research and advocacy efforts for PDCD. Hope for PDCD has quickly grown into a collective of volunteer parent and patient board members, scientific advisors, and industry partners. Hope for PDCD aims to fund a multi-million-dollar research project into AAV9 gene replacement therapy for PDHA1 mutations while pursuing equitable access to diagnosis, care and treatments for PDCD patients. Find out more at hopeforpdcd.org.
About The Elizabeth Watt PDCD Research Fund
Through outreach, philanthropy and advocacy, The Elizabeth Watt PDCD Research Fund will support research for Pyruvate Dehydrogenase Complex Deficiency, by promoting small molecule research, advocating for adding PDCD to the Newborn Screening Test, providing essential education to caregivers, and travel grants to families seeking specialized PDCD care. Learn more at pdcdresearchfund.com.
Media Contact
Andy Dearth, United Mitochondrial Disease Foundation, 1 412-744-1055, [email protected], www.umdf.org
SOURCE United Mitochondrial Disease Foundation
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