Saol Therapeutics News

Saol Therapeutics Receives Fast Track Designation for Treatment of Rare Mitochondrial Disease – Pyruvate Dehydrogenase Complex Deficiency (PDCD)

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, February 12, 2024 – Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the FDA has granted Fast Track Designation for Dichloroacetate (DCA, SL-1009) for the treatment of an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD)1.

Fast Track designation is granted to expedite the development and review of drugs that treat serious conditions and fill an unmet medical need. DCA (SL-1009) has demonstrated potential to address such needs in PDCD2,3.  Fast Track accelerates the availability of promising therapies to patients in need.

"We are pleased to receive Fast Track Designation from the FDA for SL-1009 for the treatment of PDCD," said Dave Penake, CEO of Saol Therapeutics. "This designation allows for submission of the NDA as Rolling Review. DCA (SL-1009) already has Orphan Drug Designation, as well as Rare Pediatric Disease Designation and is thus eligible for Priority Review and a Priority Review Voucher. We are hoping to file our NDA in 2024.   PDCD is a debilitating progressive disease4.  Such designations provide expedited pathways to enable us to bring this much-needed therapy to patients more swiftly."

"PDCD is a devastating childhood disease, characterized by progressive neurological and neuromuscular deterioration, lactic acidosis and early death4. Receiving Fast Track designation from the FDA is a testament to the dedication and collaborative efforts of our colleagues over many years and underscores the urgency and potential impact of our work in PDCD” notes Dr. Peter W. Stacpoole, Professor of Medicine at the University of Florida and Principal Investigator of the DCA/PDCD trial.  “We hope our DCA (SL-1009) NDA will be reviewed expeditiously and, when approved, will bring this drug to the children and families who have been long-waiting for an effective therapy.”

About the Phase 3 Clinical Trial

This trial evaluated daily administration of DCA in children with a confirmed pathological mutation in the Pyruvate Dehydrogenase Complex. The primary efficacy outcome measure was based on a novel Observer Reported Outcome (ObsRO) survey developed specifically for this trial.

The study design was a 9-month double-blind crossover comparison between DCA and placebo, during which the ObsRO is completed daily by a parent/caregiver, followed by an open-label phase of DCA administration.   The last patient completed the double-blind portion of the trial in August of 2023.  The majority of participants are currently in the open-label phase. More information on the clinical trial and participating institutions can be found at Phase 3 PDCD Trial (NCT02616484).

About DCA (SL-1009) 

DCA, if approved by the FDA after a review of safety and efficacy, has the potential to be the 1st approved medication for the mitochondrial disease PDCD and would be available as an oral solution.  Gene mutations in the mitochondrial Pyruvate Dehydrogenase Complex (PDC) lead to congenital PDCD. PDC is inhibited by pyruvate dehydrogenase kinases (PDK), that may be over-expressed in PDCD. DCA inhibits PDK to stimulate residual PDC activity and increase energy (ATP) production by mitochondria. Dosing is based on a proprietary genetic test that dichotomizes subjects into “fast” and “slow” drug metabolizers, providing individualized dosing.

About Pyruvate Dehydrogenase Complex Deficiency (PDCD)

PDCD is a mitochondrial disorder of carbohydrate oxidation that mostly affects the nervous system and skeletal muscle and leads to decreased ATP production and energy failure.  It affects several hundred individuals in the U.S.  It is a common cause of congenital lactic acidosis, a life-threatening condition that may occur as early as the neonatal period. Patients may also exhibit extreme tiredness (lethargy), poor feeding, rapid breathing (tachypnea), and other signs of neurological and neuromuscular dysfunction such as developmental delay, low muscle tone (hypotonia), abnormal eye movements and seizures. Signs and symptoms usually begin soon after birth but may appear later in childhood4.

There are currently no FDA-approved therapies for PDCD.

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


  1. 1., Accessed February 2024
  2. 2. Stacpoole PW, Gilbert LR, Neiberger RE, Carney PR, Valenstein E, Theriaque DW, Shuster JJ. Evaluation of long-term treatment of children with congenital lactic acidosis with dichloroacetate. Pediatrics. 2008 May;121(5):e1223-8. doi: 10.1542/peds.2007-2062. Epub 2008 Apr 14. PMID: 18411236; PMCID: PMC3777225.
  3. 3. Stacpoole PW, Kerr DS, Barnes C, Bunch ST, Carney PR, Fennell EM et al. Controlled Clinical Trial of Dichloroacetate for Treatment of Congenital Lactic Acidosis in Children. Pediatrics. 17(5);1519-1531, 2006.
  4. 4. Ganetzky R, McCormick EM, Falk MJ. Primary Pyruvate Dehydrogenase Complex Deficiency Overview. 2021 Jun 17. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2023. PMID: 34138529.