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 genetically defined 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.

Participating in Clinical Studies of Investigational Medicines

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

Agios Expanded Access Program

When patients with a serious or life-threatening illness have exhausted all treatment options and are not eligible for any appropriate clinical trials, they may be eligible for treatment with an investigational medicine through programs referred to as “Expanded Access Programs” (EAPs) or “compassionate use.” These programs help provide patients with a treatment that they would otherwise not be able to receive.

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.

Agios intends to work in cooperation with local health authorities and in accordance with our criteria for an EAP to provide access to our investigational medicines.


For more information about ongoing clinical
trials, please visit

For more information about Agios’ EAPs, please

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.

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

  • Agios must be studying the requested investigational medicine as part of a clinical trial, or the trial may have recently ended. Once the medicine is approved by the local health authority, 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.
  • 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. Whenever possible, patients requesting access to an Agios investigational medicine will be referred to an ongoing clinical trial.
  • 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 consultation 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.
  • There must be a 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.

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 or 833-228-8474. 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.

If approved for an EAP, your physician will work with you to address all logistical elements.