Since the beginning, we have been focused on patient needs across a wide variety of diseases. The guidance, collaboration and expertise of the patient communities we serve play a critical role in our success.

We believe in maintaining open and direct communications with the patient community. We work with partners, collaborators, organizations, and societies with similar goals to make sure patients are getting the support and resources they need. The following are some of our partners.

Ionis Pharmaceuticals is driven by our belief that sick people depend on us.  Our mission is to develop first-in-class and best-in-class medicines that transform the lives of those affected by severe diseases by improving their health and well-being.  We are committed to creating new, safe and effective medicines to treat diseases where no other therapeutic approaches have proven effective

We believe that the goal of making life-changing medicines available to the patient community is achieved through rigorous science, innovation, a commitment to patients in need, and well-designed clinical studies followed by regulatory approval.

The best way to access an investigational product, i.e. a medicine which has not yet received regulatory marketing approval, is through participation in one of our clinical studies.

We understand that there are seriously ill patients with life-threatening diseases who will not be eligible for our clinical studies and for which no approved medicine is available. Outside of a clinical study expanded access (also referred to as “compassionate use”) to investigational products will be considered, with the primary purpose of supporting those patients with a serious or immediately life-threatening condition who have exhausted all available medical options and are unable to enroll in any ongoing clinical study.

Criteria Used for Considering Requests for Expanded Access

To obtain Expanded Access from an investigational Ionis medicine, all of the following conditions must be met:

• The patient has a serious or immediately life-threatening disease or condition.
• There is no comparable or satisfactory alternative therapy for the disease or condition.
• The patient does not qualify to participate in any ongoing clinical study sponsored by Ionis, which includes lack of access due to geographic limitations.
• Sufficient clinical evidence of safety and effectiveness for the indication has been established and concluded that the potential benefit outweighs the potential risks to the patient requesting the medicine and to other patients.
• Product is under active development for the requested disease indication and making the 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 product exists.
• There is a regulatory mechanism in the country or region to support expanded access.

Process for Requesting Expanded Access

Patients interested in seeking expanded access to an Ionis investigational product should talk to their physician. A treating physician may request information about applying for access to one of Ionis’ investigational products by contacting Ionis by email at [email protected].  Confirmation of receipt of written requests will be made within five (5) working days. Decisions will be made as quickly as possible.  Ionis will carefully review all expanded access requests in consideration of the above criteria and decisions will be made as quickly as possible.

Patients and caregivers seeking general information may reach out to Ionis’ Patient Advocacy & Engagement Department at [email protected].