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FDA Issues CRL to Saol Therapeutics for PDCD Treatment
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Key Takeaways
- Saol Therapeutics received a CRL from the FDA for SL1009 (DCA) for treating PDCD, despite prior favorable designations.
- Addressing the CRL's issues will be costly and time-consuming, but Saol remains committed to working with the FDA.
The company says addressing the issues raised in the CRL could take years.
FDA issued a CRL to Saol Therapeutics for SL1009 (DCA).
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FDA issued a complete response letter (CRL) to Saol Therapeutics for SL1009 (DCA), a treatment for pyruvate dehydrogenase complex deficiency (PDCD).1
Prior to this, FDA granted the drug the following designations:
- Orphan Drug
- Priority Review
- Rare Pediatric Disease Designations
According to Saol, fewer than 1,000 people in the United States are living with PDCD. The condition impacts an estimated 90 newborns each year. The disease causes serious health problems, and a significant number of children do not survive past early childhood.
Why did FDA issue the CRL?
In a press release addressing the CRL, Saol Therapeutics CEO Dave Penake said, “Families living with PDCD face an urgent and life-threatening unmet need. We are encouraged by recent FDA actions and commentary that recognize the importance of regulatory flexibility and speed for treating rare diseases. Traditional clinical trials often take many years; time that children with PDCD simply do not have. We remain committed to productive conversations with the Agency to identify a path forward that brings this therapy to patients as swiftly as possible."
Saol did not provide the specific reasons provided by FDA for the issuance of the CRL, but the company does say that the issues are not related to the manufacturing of the drug. The company also says that addressing the issues laid out in the CRL would take several years and would be very costly.
The letter appears to have come as a surprise to Saol, who says that it stands by the clinical data collected over several years. In April of this year, the company published a press release detailing its various development programs, and included DCA among that list.2 At the time, Penake said of DCA, “We are working with the FDA to secure approval for this product this year. Our entire organization is united in the mission to bring hope to patients and families who have been waiting for this breakthrough therapy."
At the time, Saol even spoke of advancing an expanded access program for PDCD patients who were unable to participate in the trials. The company also spoke about preparations for a Type C meeting with FDA for the use of DCA to treat congenital lactic acidosis.
In January of this year, Saol announced FDA’s acceptance of its new drug application for DCA.3 At the time, Penake said, “"This NDA acceptance brings us one step closer to addressing the critical health challenges faced by these children and their families, where no approved treatment is currently available. Reaching this regulatory milestone is both a significant scientific achievement and a deeply meaningful moment for everyone who has dedicated themselves to advancing this therapy. Further, designating our application for priority review reaffirms that the FDA views PDCD as a serious condition"
While Saol decides its next moves with DCA, it’s also still moving forward with other programs in its pipeline. This includes SL1002, an injectable treatment for chronic knee pain.
In its April press release,2 Saol said of SL 1002, “"SL1002 continues to be a program with enormous potential. Our recent FDA End of Phase II meeting provided a clear regulatory path for our osteoarthritis pain program. We are eager to initiate our pivotal program this year. Meanwhile, our spasticity data was recently presented by our investigators and received positive feedback from thought leaders in the field. As we further explore SL1002's therapeutic potential, it is becoming increasingly clear that this program has far-reaching possibilities to treat patients with a variety of neuromuscular disorders."
Sources
- Saol Therapeutics Receives Complete Response Letter from FDA for SL1009 (DCA) for the Treatment of Pyruvate Dehydrogenase Complex Deficiency (PDCD). Saol Therapeutics. September 8, 2025. https://www.prnewswire.com/news-releases/saol-therapeutics-receives-complete-response-letter-from-fda-for-sl1009-dca-for-the-treatment-of-pyruvate-dehydrogenase-complex-deficiency-pdcd-302548522.html
- Saol Therapeutics Announces Major Clinical Milestones Across Multiple Development Programs. Saol Therapeutics. April 17, 2025. Accessed September 8, 2025. https://www.prnewswire.com/news-releases/saol-therapeutics-announces-major-clinical-milestones-across-multiple-development-programs-302431503.html
- Saol Therapeutics announces FDA acceptance of New Drug Application for SL1009 for treatment of Pyruvate Dehydrogenase Complex Deficiency. Saol Therapeutics. January 28, 2025. Accessed September 8, 2025. https://www.prnewswire.com/news-releases/saol-therapeutics-announces-fda-acceptance-of-new-drug-application-for-sl1009-for-treatment-of-pyruvate-dehydrogenase-complex-deficiency-302361297.html
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