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 for the inclusion of eligible patients.

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, health authorities may grant permission for Scholar Rock to provide a treating physician with an investigational therapy pre-approval outside of a clinical trial. The pre-approval access is called expanded access, or compassionate use.

Scholar Rock has an active Expanded Access Program (EAP) in the US for our investigational product, apitegromab (NCT06877689) for the treatment of eligible patients with SMA. More information about this program can be found here.

Expanded access treatment can only be provided under limited circumstances, meaning that Scholar Rock will not be able to fulfill all requests. Requests for expanded access to investigational therapies must be made by a qualified physician on the patient’s behalf. In compliance with health authority regulations, patients themselves cannot make a request for treatment and are advised to discuss with the treating physician.

For additional Information about Scholar Rock’s clinical trials and EAPs, including Expanded Access Treatment Protocols and Scholar Rock’s investigational medicines, please contact our Medical Information team at medicalinformation@scholarrock.com or 1-855-MED-SRRK.