Scholar Rock’s Expanded Access Policy

Date of Last Revision: May 7, 2019

Scholar Rock is a biopharmaceutical company focused on the discovery and development of safe and effective therapies for a diverse range of serious diseases, including neuromuscular disorders, cancer, fibrosis, and anemia.  As we advance our investigational therapies through clinical development, our goal is to provide access to these therapies at the appropriate time and in the correct manner for patients.

Expanded access, or “compassionate use”, refers to a potential pathway for a patient with a serious or life-threatening disease or condition to try an investigational therapy outside a clinical trial when there is no comparable or satisfactory therapy available.  Several factors consistent with guidelines by the U.S. Food and Drug Administration (FDA) and other regulatory agencies should be taken into account when considering expanded access, including:

  • 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, Scholar Rock does not have an investigational product candidate available for expanded access.  We believe that participation in clinical trials, which are carefully designed to determine the safety and efficacy of an investigational therapy, is the most appropriate way for patients to access an investigational therapy until sufficient evidence is available that suggests the potential benefit outweighs the risk.

For questions regarding our expanded access policy, please contact  We anticipate acknowledging receipt of any expanded access questions within five business days following receipt.

Scholar Rock may revise this expanded access policy at any time and will provide any updates to the policy on our website.

“Scholar Rock” is a registered trademark of Scholar Rock, Inc.  |  © 2020 Scholar Rock All Rights Reserved.
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