Saol Therapeutics Pursues FDA Approval for SL1009 in Treating Rare Genetic Disorder

Saol Therapeutics Pursues FDA Approval for SL1009

Saol Therapeutics, a clinical-stage pharmaceutical company, is currently navigating challenges following the U.S. Food and Drug Administration's (FDA) issuance of a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for SL1009, or Sodium Dichloroacetate (DCA). This investigational therapy is aimed at treating Pyruvate Dehydrogenase Complex Deficiency (PDCD), a rare and serious genetic disorder affecting young children.

This debilitating condition is estimated to impact fewer than 1,000 individuals within the United States and has an incidence rate of approximately 1 in every 40,000 live births, which translates to around 90 diagnosed cases each year. Unfortunately, children diagnosed with PDCD often face severe health complications, with many not surviving infancy. As of now, there are no FDA-approved treatments available for PDCD, making SL1009 a potential lifeline for affected families.

In the recently received CRL, the FDA outlined specific observations which Saol must address in order to outline a clear path for moving forward with the application. Importantly, these observations did not point to any manufacturing deficiencies with SL1009. However, addressing the issues raised will likely take several years and necessitate substantial financial investment. Despite this setback, Saol remains encouraged by the data derived from four years of clinical research, which includes two phase III trials — one of which is an ongoing double-blind, placebo-controlled study that has progressed into an open-label extension phase. Furthermore, the company conducted survival analyses against natural history cohorts that indicate the potential benefits of SL1009.

Dave Penake, CEO of Saol Therapeutics, emphasized the dire need faced by families with children suffering from PDCD, stating, "Families living with PDCD face an urgent and life-threatening unmet need. We are encouraged by recent FDA actions and commentary that reflect the necessity of regulatory flexibility and expeditious measures for dealing with rare diseases. Traditional clinical trials typically consume considerable time, which is untenable for children affected by PDCD."

The company continues to work closely with the FDA to find a resolution that will facilitate bringing SL1009 to patients as quickly as possible. In the meantime, Saol assures that access to SL1009 remains available through the ongoing open-label extension of the clinical trial (ClinicalTrials.gov Identifier: NCT02616484) as well as their Expanded Access Program (ClinicalTrials.gov Identifier: NCT06931262), the latter also providing emergency support for newborns suffering from life-threatening lactic acidosis due to inherited metabolic disorders.

Saol Therapeutics has also maintained a focus on the urgent healthcare needs of the PDCD community and is committed to collaborating with the FDA to reach a favorable conclusion for the SL1009 application. Updates will be shared in due course as dialogues progress.

About SL1009, Sodium Dichloroacetate (DCA)

SL1009 is being developed as an investigational medication intended for use alongside a proprietary genetic test that will assist in determining dosages for patients with PDCD. Given the condition's rarity and potentially fatal implications — which can lead to chronic energy deficits, developmental issues, and early death — SL1009's approval could mark a critical advancement in treatment options for affected children.

The therapy has already secured designations from the FDA including Priority Review, Orphan Drug Designation, and Rare Pediatric Disease Designation, underscoring its significance in addressing a dire need. Saol, in partnership with Medosome Biotec, has also submitted a Humanitarian Device Exemption (HDE) application for the associated genetic test that will serve as a mandatory diagnostic tool for patients treated with SL1009.

About Saol Therapeutics

Founded with a focus on developing treatments for Central Nervous System (CNS) disorders, pain management, and orphan diseases, Saol Therapeutics is a privately held company with operations in Roswell, Georgia; Dublin, Ireland; and Hamilton, Bermuda. Saol is dedicated to advancing therapeutic options for patients and their healthcare providers. For more information, visit www.saolrx.com.