FDA grants a Type A meeting on December 18th to review new and expanded datasets submitted by Saol to help identify a feasible path forward without the need for an additional clinical trial
ROSWELL, Ga., DUBLIN and HAMILTON, Bermuda — December 9, 2025 — Saol Therapeutics, a privately held clinical-stage pharmaceutical company, today announced that the company has been granted a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss new data and a potential path forward for its New Drug Application (NDA) for SL1009, Sodium Dichloroacetate Oral Solution (DCA) for use with a proprietary genetic test, for the treatment of an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). SL1009 has been granted Orphan Drug, Priority Review, and Rare Pediatric Disease Designations by the FDA.
In the two months since receiving an unexpected Complete Response Letter (CRL), Saol has collected additional data and performed new analyses to help address the clinical deficiencies raised by the FDA. Saol’s objective is to collaborate with the FDA on a pathway to approval without the need for a new clinical trial, which is not feasible.
“The FDA’s recent commitments to clearer, more efficient pathways for rare disease drug development and to ensuring science and efficacy-driven decision-making are encouraging,” said Dave Penake, chief executive officer of Saol Therapeutics. “For an ultra-rare condition like PDCD, the challenge is navigating a regulatory process not built for such small patient populations. We remain optimistic given the strength of the data to date, the years of clinical observation supporting DCA, and the Agency’s renewed focus on modernizing rare disease approval processes through programs like the Rare Disease Evidence Principles (RDEP) so that families aren’t forced to endure unnecessary delays.”
At the upcoming Type A meeting, Saol will present new and expanded analyses not previously reviewed by the Agency, including:
- Functional Benefit: Additional analyses of functional benefit data, including longer treatment duration.
- Survival Benefit: Saol obtained additional data on the comparability of the natural history cohort and the treatment cohort, with new analyses of survival benefit.
- Mechanistic Support: Further supporting evidence that SL1009 directly acts to correct the underlying enzymatic abnormality caused by PDCD.
- Safety: Over one hundred patient-years of exposure and many patients with over four years of therapy continue to support the well-established safety profile of SL-1009.
Saol believes these data reinforce the risk/benefit of SL1009 and demonstrate how the totality of evidence satisfies the FDA’s proposed Rare Disease Evidence Principles.
Penake added, “We’re approaching this next step with optimism and a spirit of collaboration. With the new analyses we’ve compiled, we are seeking a path forward without conducting an additional clinical trial. Our goal is to align on a science-driven solution that supports families facing this devastating disease, which currently has no approved treatment options.”
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.
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.
Media Contact:
Anna Stallmann
media@ascentcomms.com
In the two months since receiving an unexpected Complete Response Letter (CRL), Saol has collected additional data and performed new analyses to help address the clinical deficiencies raised by the FDA. Saol’s objective is to collaborate with the FDA on a pathway to approval without the need for a new clinical trial, which is not feasible.
“The FDA’s recent commitments to clearer, more efficient pathways for rare disease drug development and to ensuring science and efficacy-driven decision-making are encouraging,” said Dave Penake, chief executive officer of Saol Therapeutics. “For an ultra-rare condition like PDCD, the challenge is navigating a regulatory process not built for such small patient populations. We remain optimistic given the strength of the data to date, the years of clinical observation supporting DCA, and the Agency’s renewed focus on modernizing rare disease approval processes through programs like the Rare Disease Evidence Principles (RDEP) so that families aren’t forced to endure unnecessary delays.”
At the upcoming Type A meeting, Saol will present new and expanded analyses not previously reviewed by the Agency, including:
- Functional Benefit: Additional analyses of functional benefit data, including longer treatment duration.
- Survival Benefit: Saol obtained additional data on the comparability of the natural history cohort and the treatment cohort, with new analyses of survival benefit.
- Mechanistic Support: Further supporting evidence that SL1009 directly acts to correct the underlying enzymatic abnormality caused by PDCD.
- Safety: Over one hundred patient-years of exposure and many patients with over four years of therapy continue to support the well-established safety profile of SL-1009.
Saol believes these data reinforce the risk/benefit of SL1009 and demonstrate how the totality of evidence satisfies the FDA’s proposed Rare Disease Evidence Principles.
Penake added, “We’re approaching this next step with optimism and a spirit of collaboration. With the new analyses we’ve compiled, we are seeking a path forward without conducting an additional clinical trial. Our goal is to align on a science-driven solution that supports families facing this devastating disease, which currently has no approved treatment options.”
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.
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.
Media Contact:
Anna Stallmann
media@ascentcomms.com