In the spirit of transparency, Saol publicly details FDA’s Complete Response Letter and next steps for DCA treatment in PDCD
ROSWELL, Ga, DUBLIN, and HAMILTON, Bermuda – September 19, 2025 – On September 18, 2025, Saol Therapeutics presented a detailed overview in the Hope for PDCD public town hall to address the recent Complete Response Letter (CRL) issued by the FDA for SL1009 (Sodium Dichloroacetate, DCA), an investigational therapy for Pyruvate Dehydrogenase Complex Deficiency (PDCD).
During the webinar, Saol leadership outlined the FDA’s decision, shared trial data, discussed potential next steps, and reaffirmed their commitment to pursuing a regulatory path forward that does not require an additional trial. Families also shared firsthand stories of what life with PDCD is like, underscoring the urgent need for treatment options.
WATCH THE RECORDING:
Watch the webinar recording at this link.
WHO:
During the webinar, Saol leadership outlined the FDA’s decision, shared trial data, discussed potential next steps, and reaffirmed their commitment to pursuing a regulatory path forward that does not require an additional trial. Families also shared firsthand stories of what life with PDCD is like, underscoring the urgent need for treatment options.
WATCH THE RECORDING:
Watch the webinar recording at this link.
WHO:
- Dave Penake – CEO, Saol Therapeutics
- Kyle Ashton, PhD – Head of Medical Affairs, Saol Therapeutics
- Sharon Hamm, PharmD, RPH – Chief Development Officer, Saol Therapeutics
- Brian Nappi – Chief of Strategy, Saol Therapeutics
- Families and advocates from Hope for PDCD and the greater mitochondrial community
KEY POINTS DISCUSSED:
- FDA’s CRL for SL1009 did not cite any manufacturing or safety issues; instead, it called for clarifications that, if pursued through another traditional trial, could take years and significant resources
- Saol reaffirmed continued access to SL1009 through both the open-label extension trial and the Expanded Access Program
- Saol is committed to working with FDA on a solution that accelerates patient access, citing the urgent unmet need for children with PDCD who “simply do not have time” for lengthy trial delays
- Hope for PDCD shared patient and caregiver perspectives, highlighting the daily impact of PDCD on families, and the shortcomings of a ketogenic diet as the current standard of care
WHY IT MATTERS:
- PDCD affects fewer than 1,000 individuals in the U.S., with an incidence estimated at 1 in 40,000 live births, approximately 90 newborns annually1
- There are currently no FDA-approved therapies for PDCD
- Saol’s openness in addressing the CRL publicly is unusual in drug development and underscores both the urgency and transparency needed in rare disease innovation
About SL1009, Sodium Dichloroacetate (DCA)
SL1009 is an investigational product that, if approved, will be used with a proprietary dose-determining genetic test to treat an orphan pediatric-onset mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). PDCD is a rare and life-threatening genetic disorder that can cause chronic energy deficit leading to lactic acidosis, profound developmental problems, and early childhood death. There are currently no FDA-approved therapies for PDCD.
SL1009 has received Priority Review, Orphan Drug Designation, and Rare Pediatric Disease Designation. Saol, in collaboration with Medosome Biotec, also filed the Humanitarian Device Exemption (HDE) application for the dose-determining genetic test that will serve as a required companion diagnostic for patients treated with SL1009.
About Saol Therapeutics
Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland, and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit www.saolrx.com.
Saol Therapeutics Contact
Anna Stallmann
media@ascentcomms.com
1 Bedoyan JK. Pyruvate Dehydrogenase Complex Deficiency. Medlink Neurology. 2024 May; https://www.medlink.com/articles/pyruvate-dehydrogenase-complex-deficiency
SL1009 is an investigational product that, if approved, will be used with a proprietary dose-determining genetic test to treat an orphan pediatric-onset mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). PDCD is a rare and life-threatening genetic disorder that can cause chronic energy deficit leading to lactic acidosis, profound developmental problems, and early childhood death. There are currently no FDA-approved therapies for PDCD.
SL1009 has received Priority Review, Orphan Drug Designation, and Rare Pediatric Disease Designation. Saol, in collaboration with Medosome Biotec, also filed the Humanitarian Device Exemption (HDE) application for the dose-determining genetic test that will serve as a required companion diagnostic for patients treated with SL1009.
About Saol Therapeutics
Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland, and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit www.saolrx.com.
Saol Therapeutics Contact
Anna Stallmann
media@ascentcomms.com
1 Bedoyan JK. Pyruvate Dehydrogenase Complex Deficiency. Medlink Neurology. 2024 May; https://www.medlink.com/articles/pyruvate-dehydrogenase-complex-deficiency