Ensuring patients get the information they need

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 Clinical Trials

Before any treatment is approved for human use by the FDA and other regulatory authorities around the world, it must first prove to be safe and effective in clinical trials.

Ionis clinical trials

Our trials are designed with the greatest needs of the patients who will benefit from them in mind. They reflect input from patient communities as well as healthcare providers and regulatory agencies who share our commitment to providing access to safe and effective therapies as soon as possible.

We have many ongoing clinical trials designed to evaluate our medicines in a variety of areas of unmet medical need, including cardiovascular, metabolic, severe and rare diseases, including neurological disorders and cancer.

Learn more about Ionis clinical trials


For answers to questions you may have, connect with us.

Expanded Access Policy

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].

Three smiling boys with cure sma tshirts on

Effecting change through community

In our pursuit to create a brighter future for patients, we must partner and collaborate to change the healthcare landscape, increase disease awareness and drive innovation.

See Ionis’ community engagement

Male and Female in lab smiling

Once in a lifetime breakthroughs again and again

Meet Sonia and Eric. When genetic testing revealed that Sonia would ultimately suffer from the same fatal prion disease as her mother, Sonia and her husband, Eric, refused to sit back and let the disease ravage their family.

See who inspires Ionis