Expanded Access

Scholar Rock is committed to creating new possibilities for people living with serious disease. As we advance multiple investigational therapies through clinical development, our goal is to provide timely and appropriate access to therapies with life-changing potential.

Patients generally obtain access to investigational therapies prior to regulatory approval by participating in clinical trials. We are purposeful in designing inclusive clinical trials that are consistent with regulatory guidance and include as many eligible patients as possible. More information about Scholar Rock’s clinical trials can be found here.

However, there may be instances where a patient with a serious or life-threatening disease or condition is not eligible to participate in a clinical trial and has exhausted all treatment options. In these cases, patients may be eligible for treatment with an investigational therapy outside of a clinical trial under expanded access, or “compassionate use.”

We understand the patient interest in accessing investigational therapies outside of a clinical trial. If expanded access is allowed by local laws and regulation, additional criteria are often required for a patient to receive treatment through expanded access, such as:

• The illness must be serious or life-threatening with no other satisfactory treatment options (such as approved products or enrolling clinical trials involving the eligible investigational drug).
• There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on available safety and efficacy information.
• The patient’s clinician has requested access to the investigational therapy.
• The decision to grant expanded access does not compromise broader development programs or interfere with or delay 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.