Expanded Access Program Policy

Expanded Access Treatment with Sodium Dichloroacetate (DCA) for Patients with Pyruvate Dehydrogenase Complex Deficiency (PDCD)

Expanded access is a potential pathway for patients diagnosed with PDCD to gain access to DCA, an investigational treatment, for treatment outside of a clinical trial when no comparable or satisfactory alternative therapy options are available. In addition, Saol’s Expanded Access Program allows for a transition to continued therapy in patients who are currently enrolled in the Open Label Extension of the Phase III study SL1009-01. Saol’s Expanded Access Program is managed by the company and follows a specific protocol.

Additional information about this Expanded Access Program can be found on Clinicaltrials.gov,  NCT06931262.

 

Eligibility for the PDCD Expanded Access Program

Saol considers granting expanded access to DCA when all of the following criteria are met:

  • A patient is diagnosed with PDCD, and no comparable or satisfactory alternative therapy is available to treat the disease or condition.
  • The patient is not eligible to participate in a clinical trial.
  • The patient’s licensed physician (“physician”) submits an unsolicited request for expanded access.
  • Sufficient data exists where the potential benefits of the treatment outweigh the potential risks for the disease to be treated.
  • There is adequate supply of the investigational product, and providing the investigational product will not interfere with any ongoing clinical trials or the overall development program.
  • Investigational access provided in this setting is allowed per applicable law.

How to Request Access for the PDCD Expanded Access Program

We encourage patients to speak first with their physician about their eligibility for expanded access. The physician then should contact Saol to become an EAP site so patients at that site may be considered for EAP enrollment. The requesting physician must agree to obtain applicable regulatory and ethics committee approvals, and to comply with all other safety, monitoring and patient consent requirements. Patients should contact their physician with any additional questions.

Only a qualified, licensed, treating physician may submit a request for expanded access to DCA for PDCD. Please contact us at SaolEAP@anovorx.com to submit a request for expanded access.

How the Decision is Made for the PDCD Expanded Access Program

Saol has established a process for reviewing individually all requests for expanded access. Based on the medical information included in the request form submitted by the patient’s physician and any additional information needed to fully understand the patient's situation, applications will be assessed on a case-by-case basis in a fair, compassionate, and thoughtful manner. In addition, as we review requests for expanded access, we will respect patient confidentiality and comply with applicable privacy laws.

We know how important it is to receive a fast response. Patient safety and wellbeing, however, is our priority. We aim to use our best efforts to respond to most requests within 7 days of receipt of a fully completed request. However, complicated requests may require additional consultation and discussion, and this may mean we need more time.

DISCLAIMER: Treatment through expanded access can only be provided within limited circumstances and is not a guarantee of access, meaning that Saol will not be able to fulfill all requests, even when eligibility criteria are met. Patients themselves cannot make a request for treatment and always need to contact their physician.

 

Expanded Access for SL1002

Saol does not currently offer an expanded access program for treatment of patients outside of current clinical trials conducted using SL1002.

 

Additional Background

Expanded access programs are only available for specific patients with a serious or immediately life-threatening disease or condition with no other available treatment options, including clinical trials, and are not without risks.

These programs apply to medicines not yet approved by the FDA. A physician must decide whether the potential benefit outweighs the risk of receiving an investigational or unapproved medicine, based on the individual patient's medical history.

Additionally, applicable Institutional Review Board (IRB) (ethics committee that approves and monitors clinical trials involving humans) review and approvals must be obtained, including, as required, at the patient's treating hospital or clinic. As noted, expanded access programs are also subject to patient informed consent and safety reporting requirements.

Saol may revise this policy at any time.

Last updated: May 1, 2025