Ionis Expanded Access Policy

IONIS EXPANDED ACCESS POLICY

Our mission is to develop medicines that transform the lives of those affected by severe diseases by improving their health and well-being.  We are committed to creating new medicines for people where there is an unmet need.

We believe that the goal of making life-changing medicines available to the patient community is achieved through rigorous science, innovation, a commitment to people in need and well-designed clinical studies followed by regulatory approval. The best way to access an investigational medicine, i.e., a medicine being studied that has not received regulatory marketing approval, is through participation in a clinical trial.

Expanded access, also referred to as compassionate use, is one potential pathway for people with life-threatening diseases to gain early access to an investigational medicine outside of a clinical trial when no satisfactory alternative treatment is available. Ionis brought together our medical, clinical research and development, regulatory and senior leadership colleagues to thoroughly evaluate and challenge our policy on expanded access. We have further refined our position through communications with patient advocacy groups and expert independent bioethicists. In alignment with regulatory guidance and developed using the FDA criteria for expanded access, our Ionis policy considers the following criteria to determine if an Ionis investigational medicine can be made available for treatment outside of a clinical trial.

  • The individual has a serious or immediately life-threatening disease or condition.
  • There is no comparable or satisfactory alternative therapy for the disease or condition.
  • The individual does not qualify to participate in any ongoing clinical trial.
  • Sufficient clinical evidence of safety and effectiveness for the indication has been established and concluded that the potential benefit outweighs the potential risks.
  • The investigational medicine is under active development for the requested disease indication.
  • Making the investigational medicine available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access.
  • Adequate supply of investigational medicine exists.
  • There is a regulatory mechanism in the country or region to support expanded access.
     

We review and consider expanded access to our investigational medicines regularly, especially as new information about an investigational medicine and its impact emerges from our clinical trials. While reviews like this may not lead to an expanded access program allowing access outside of clinical trials, upholding our commitment to patients means ensuring decisions of this magnitude are discussed and reviewed continually. 

This site provides information on available Ionis Expanded Access Programs (EAPs) including the request process. If a specific EAP is not listed for a condition or investigational medicine, it indicates that there are no currently available EAPs from Ionis for that particular condition or investigational medicine. In these cases, participation in a clinical trial is the only way to access the investigational medicine.

It’s important to remember that investigational medicines have not yet received regulatory approval; therefore, their potential risks and benefits are not yet established. Doctors and patients should consider all possible benefits and risks when seeking expanded access to an investigational medicine. We encourage individuals to speak directly with their treating physician regarding current or future options, clinical trials, and expanded access programs. 

For questions about our investigational medicines not related to an available Ionis EAP listed below, physicians may contact Ionis directly at MedInfo@ionis.com. We anticipate acknowledging receipt of requests for information sent to this email within 5 business days. 

Some of our investigational medicines are being developed with an industry partner. Application of this Ionis Expanded Access Policy to these medications is dependent on the partnership agreement we have in place.

In line with the 21st Century Cures Act, Ionis may revise this policy at any time. 
 

Available Expanded Access Programs at Ionis

Zilganersen for Alexander Disease (AxD)

At present, Ionis Pharmaceuticals has an EAP available for zilganersen. Zilganersen is an investigational medicine designed to stop the excess glial fibrillary acidic protein (GFAP) production that accumulates because of disease-causing variants in the GFAP gene. The zilganersen EAP is currently open in the United States only, for individuals aged 2 and older living with Alexander Disease (AxD). The inclusion and exclusion criteria for the EAP are listed below. 

Inclusion Criteria 

  1. Approved drug or drugs available for treatment did not work for the patient, or the patient cannot tolerate the side effects of the FDA-approved drug or drugs for treatment of AxD.
  2. Patients who are ≥ 2 years old.
  3. Patients who have a clinical phenotype and brain imaging consistent with a diagnosis of AxD.
  4. Patients who have a documented variant in the GFAP gene.  
  5. Patient resides in and is a resident of the United States. 

Exclusion Criteria 

  1. Patients who have any medical history, physical exam findings or clinically significant laboratory abnormalities that contraindicate performing a lumbar puncture for intrathecal bolus (ITB) administration of the Product.
  2. Patients who have current obstructive hydrocephalus.
  3. Patients who have the presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF)  
  4. Patients who have any other medical history or current conditions, which, in their Investigator’s opinion, would make the Patient unsuitable for inclusion (e.g., active HCV or HBV infection, severe hepatic or renal disease, uncontrolled acute or chronic condition, etc.)  
  5. Patients who are pregnant or plan to become pregnant, or patients who are breastfeeding. 

If you are a patient, family member, or caregiver wishing to know more about this zilganersen EAP for AxD, please discuss this EAP and all treatment options with your treating physician. 

If you are a treating physician and are seeking information about the zilganersen EAP or would like to request access for a patient, please contact patient.access@bioincalemas.com.

Bionical Emas is a Clinical Research Organization managing the zilganersen EAP on behalf of Ionis.

Safety and efficacy have not been evaluated or established by any regulatory authorities for the investigational medicines and indications described above.

 

MED-US—2301035 v7 December 2025