Patients & Caregivers

About Agios’ Investigational Medicines

At Agios, we are passionately committed to applying our scientific leadership in the field of cellular metabolism to transform the lives of people with cancer and rare genetic diseases. Our focus is on discovering, developing and commercializing breakthrough medicines for the people who need them.

Before these and all other investigational medicines can be made broadly available, the U.S. Food and Drug Administration (FDA) and other health authorities around the world require that they are studied in clinical trials. Throughout this process, the safety of patients taking our medicines is of the utmost importance.

Clinical trials are research studies that are designed to determine if a medicine is safe and effective for patients.

People who choose to participate in clinical trials play a critical role in developing new medicines, particularly for rare diseases and conditions that are especially difficult to treat. Whenever possible, Agios will refer an individual requesting access to our investigational medicines to an appropriate clinical trial.

For more information about Agios’ ongoing clinical trials, visit our Clinical Trials or visit and search “Agios.” To discuss your eligibility for an Agios clinical trial, please ask your doctor to contact us on your behalf at 833-228-8474 or For information regarding Agios’ approved treatments, visit Our Medicines page.

In some cases, patients with serious or immediately life-threatening diseases may not be eligible for any clinical trials and may not have other treatment options. Generally, and only in rare and very specific circumstances, these patients may be able to receive investigational medicines outside the clinical trial process.

One way patients may be able to receive investigational medicines is through “Expanded Access Programs” (sometimes referred to as “Compassionate Use”). EAPs are for people with serious or life-threatening diseases who have exhausted all treatment options and are not eligible for any appropriate clinical trials. Generally, there are two types of EAPs: 1) those for individuals, and 2) those for groups. Agios is committed to providing access to investigational medicines in cooperation with local health authorities and in accordance with our criteria for an EAP.

These programs only apply to medicines not yet approved by the local health authorities. A doctor must decide whether the potential benefit outweighs the risk of receiving an investigational or unapproved medicine, based on the individual patient’s medical history.

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How to Request Access to an Agios Investigational Medicine

If you are interested in obtaining access to an investigational medicine, you should discuss available options with your qualified treating physician. If an Agios clinical trial may be a good treatment option for you, we recommend that your doctor contact us on your behalf at 833-228-8474 or to discuss your specific medical condition and trial eligibility requirements.

If you are not eligible for an open or pending clinical trial, or if no appropriate clinical trials exist, and if your qualified treating physician believes that an EAP may be the best or only treatment option for you, please ask your doctor to contact Agios on your behalf at 833-228-8474 or Requests will be acknowledged within 2 business days of receipt. If after contacting Agios, your doctor is supportive of and committed to the requested treatment, he/she must submit a formal request to Agios for consideration.

How Agios Makes Decisions About Expanded Access

Agios is committed to a fair and impartial evaluation of each request for access to our investigational medicines. Therefore, all decisions are based solely on clinical evidence and guided by the principles outlined below. In addition, Agios intends to work in cooperation with local health authorities for the provision of an EAP.

Agios May Consider Providing an EAP If All of These Criteria Are Met

  • Access to the Agios investigational medicine is for a patient(s) with a serious or life-threatening condition for whom no other treatment options or clinical trials are available. If and when appropriate, an EAP is a solution of last resort.
  • Agios must be studying the requested investigational medicine as part of a clinical trial. Upon commercial availability of the medicine, following local health authority approval, an EAP is no longer an option.
  • Granting access to an Agios investigational medicine must not interfere in any way with the completion of clinical trials that could support health authority approval of the medicine. Whenever possible, patients requesting access to an Agios investigational medicine will be referred to an ongoing clinical trial.
  • Patient must not be eligible for ongoing or pending clinical trials of the investigational medicine. Geographic limitations to participation in a clinical trial would generally not impact eligibility.
  • Potential benefits to the patient seeking access to the investigational medicine must outweigh the collective potential risks to the patient. Agios evaluates these benefits and risks based on case-by-case, clinical assessments in consult with the patient’s qualified treating physician.
  • Adequate clinical data must exist to support the appropriate dose amount and frequency of the investigational medicine and duration of treatment. Agios’ clinical team will determine whether sufficient clinical data is available.
  • Sufficient supply of the investigational medicine to support the EAP. Supplying the medicine through the EAP must not compromise the supply of medicine available for active or soon-opening clinical trials.