Expanded Access Policy

Concern for Patients

At Acceleron, we are committed to patient health, safety and well-being. We value the patient voice in all of our decisions, and focus our research and development on disease where we can make a difference in the lives of patients with few or no effective therapeutic options. As part of the drug development process, we work closely with patients to help us better understand how disease affects their everyday lives, which enables us to find creative solutions for bringing potentially life-changing treatments to patients faster.

We are testing medicines in clinical trials to treat the chronic, often debilitating anemias associated with serious blood disorders including myelodysplastic syndromes (MDS), beta-thalassemia and myelofibrosis. We are also conducting clinical trials in pulmonary arterial hypertension (PAH).

You can view a full list of our clinical trials, here.

Expanded Access Policy

Acceleron is dedicated to the discovery and development of innovative, life-changing medicines for patients with a wide range of serious and rare diseases. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.

Expanded access, sometimes referred to as compassionate use, refers to the use of an investigational therapy outside a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient. This is different from a clinical trial, where more comprehensive safety and efficacy data are collected. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.

A number of factors consistent with the US Food and Drug Administration (US FDA) and other regulatory agencies’ guidelines should be taken into account when considering expanded access. They include:

  • The illness must be serious or life-threatening with no other satisfactory treatment options (such as approved products or enrolling clinical trials)
  • There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on available safety and efficacy information
  • Ability to provide a product in a fair and equitable manner, so that there is adequate manufacturing capacity for ongoing programs
  • Whether granting expanded access would potentially compromise the scientific validity of broader development programs, or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients

At this time, we believe that participation in one of our clinical trials is the appropriate way to access our investigational therapies. If you have additional questions, please speak with your physician or contact We anticipate acknowledging receipt of requests sent to this email within five business days.

Acceleron may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the expanded access record on after such record becomes active.