U.S. Expanded Access Policy

BridgeBio is on a mission to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases, and is dedicated to serving these individuals, their families, and the communities they are part of through education, empathy, and awareness.

Consistent with our mission to bring innovative medicines to patients with serious or life-threatening genetic conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing. At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, BridgeBio may consider providing a requesting physician with pre-approval access to a specific investigational drug being developed by BridgeBio, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions, though not exhaustive, may include the following:

  • The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
  • The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition; 
  • Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; 
  • The requesting physician must have the Institutional Review Board (IRB) of the treating hospital or clinic review and approve the decision for the patient to use the medicine and comply with all regulatory obligations and BridgeBio requirements, including obtaining patient consent, patient monitoring, and safety reporting; 
  • A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available; and
  • Adequate supply of the investigational drug is available


We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis.

BridgeBio is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; BridgeBio may require more detailed information to fully evaluate a request. Each request will be given careful consideration by BridgeBio whose decisions are final. Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to medinfo@bridgebio.com.  BridgeBio will acknowledge receipt of requests within 5 days.