Existing data supports direct path to resubmission following constructive FDA engagement
ROSWELL, Ga., DUBLIN and HAMILTON, Bermuda — July 7, 2026 — Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, today announced the resubmission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for SL1009, sodium dichloroacetate (DCA), for the treatment of pyruvate dehydrogenase complex deficiency (PDCD), a rare, life-threatening mitochondrial disease with no FDA-approved therapies.
The resubmission follows a Type A meeting in December and a Type C meeting in March, during which the FDA provided guidance recommending additional survival analyses to support the application. This guidance enabled Saol to proceed directly to resubmission without conducting an additional trial, an outcome Saol believes reflects the FDA’s commitment to working constructively with sponsors to identify viable regulatory paths for programs serving patients with serious unmet needs.
Saol’s resubmission follows the receipt of a Complete Response Letter (CRL) in August 2025, which did not identify concerns related to safety or manufacturing but requested additional evidence to support approval. In response, Saol conducted further analyses of existing data, including functional outcomes, survival, mechanistic evidence, and long-term safety data.
“We are encouraged by the clarity and alignment achieved with the FDA during our recent interactions,” said Dave Penake, chief executive officer of Saol Therapeutics. “This resubmission reflects the totality of evidence supporting SL1009. We are committed to continuing to work collaboratively with the Agency and are encouraged by the FDA’s demonstrated willingness to apply regulatory flexibility.”
Penake added, “We are grateful for the continued engagement of the PDCD community and experts, whose efforts have helped communicate the impact of this disease on affected individuals and families. We remain focused on advancing our NDA resubmission while continuing to support the need for timely evaluation of therapies for rare, life-threatening conditions.”
SL1009 has been studied across multiple clinical trials, including two Phase 3 studies and long-term open-label extension data, supporting a well-characterized safety profile. Saol believes the totality of evidence supports the potential for meaningful clinical benefit in patients with PDCD. The FDA will assign a new action (PDUFA) date upon acceptance of the file.
SL1009 has received Priority Review, Orphan Drug Designation, and Rare Pediatric Disease Designation, and Saol anticipates receiving a Priority Review Voucher (PRV) under the Rare Pediatric Disease Statute.
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.
Media Contact
Mae Young
media@ascentcomms.com
The resubmission follows a Type A meeting in December and a Type C meeting in March, during which the FDA provided guidance recommending additional survival analyses to support the application. This guidance enabled Saol to proceed directly to resubmission without conducting an additional trial, an outcome Saol believes reflects the FDA’s commitment to working constructively with sponsors to identify viable regulatory paths for programs serving patients with serious unmet needs.
Saol’s resubmission follows the receipt of a Complete Response Letter (CRL) in August 2025, which did not identify concerns related to safety or manufacturing but requested additional evidence to support approval. In response, Saol conducted further analyses of existing data, including functional outcomes, survival, mechanistic evidence, and long-term safety data.
“We are encouraged by the clarity and alignment achieved with the FDA during our recent interactions,” said Dave Penake, chief executive officer of Saol Therapeutics. “This resubmission reflects the totality of evidence supporting SL1009. We are committed to continuing to work collaboratively with the Agency and are encouraged by the FDA’s demonstrated willingness to apply regulatory flexibility.”
Penake added, “We are grateful for the continued engagement of the PDCD community and experts, whose efforts have helped communicate the impact of this disease on affected individuals and families. We remain focused on advancing our NDA resubmission while continuing to support the need for timely evaluation of therapies for rare, life-threatening conditions.”
SL1009 has been studied across multiple clinical trials, including two Phase 3 studies and long-term open-label extension data, supporting a well-characterized safety profile. Saol believes the totality of evidence supports the potential for meaningful clinical benefit in patients with PDCD. The FDA will assign a new action (PDUFA) date upon acceptance of the file.
SL1009 has received Priority Review, Orphan Drug Designation, and Rare Pediatric Disease Designation, and Saol anticipates receiving a Priority Review Voucher (PRV) under the Rare Pediatric Disease Statute.
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.
Media Contact
Mae Young
media@ascentcomms.com