Expanded Access

Policy on Expanded Access

for Investigational Medicines

Expanded Access

Orca Bio is a company focused on developing innovative therapies for patients with serious, life-threatening disease. In our mission to bring these treatments to patients as quickly as possible, we conduct clinical trials to establish the safety and efficacy of an investigational product, which is a medicine in active clinical development but one that has not yet fulfilled all data requirements and/or received marketing approval by the relevant health authorities, such as the FDA.

In our mission to bring treatments to patients as quickly as possible, we conduct clinical trials to establish the safety and efficacy of an investigational product.

Clinical trials are a well-established modality for the generation of data as a necessary step in bringing innovative, safe and effective medicines to patients in need of improved treatment options. For this reason, we prioritize access to an investigational medicine through clinical trials and encourage patients to enroll in such studies. More information can be found at: www.clinicaltrials.gov.

However, we recognize that there are circumstances where patients with serious or life-threatening diseases may not be eligible for, or are able to participate in, one of our trials. In such circumstances, subject to the criteria set forth below and country specific regulations, patients may be eligible to access Orca Bio’s investigational therapies through our Expanded Access Policy. This document serves to outline our policy on expanded access, also referred to as compassionate use, early access, pre-approval access and emergency use programs – all are special access to an investigational therapy outside of a clinical trial setting. Expanded access programs for any investigational medicine will terminate once regulatory (i.e., marketing) approval is granted, thereby authorizing its general availability for physicians and patients in the jurisdiction of the regulatory agency.

Orca Bio’s ethical responsibilities ensure the quality and integrity of clinical trials and compassionate use to minimize risks to research participants and future patients. Therefore, a request for compassionate use of an investigational medicine must meet all the following requirements:

  • Use of any investigational medicine must be overseen by a licensed and appropriately qualified physician, in accordance with local laws and regulations governing such programs, as well as all Orca policies and procedures.
  • Orca Bio has determined that sufficient preliminary efficacy and safety data have been generated to identify an appropriate dose and that the benefit-risk analysis is consistent with a compassionate use program (i.e. that any potential benefits from treatment are likely to outweigh any associated risks to the patient ).
  • The patient does not qualify for participation in ongoing or pending clinical trials and all necessary regulatory and institutional approvals have been obtained to allow administration of the investigational product.
  • The disease or condition is life-threatening and is commensurate with the indication for which the investigational therapy is likely to be approved.
  • Supply logistics for the treatment can be achieved such that the therapy can be provided in a timely and effective manner.
  • Treating under an expanded access program will not adversely impact, impede, or compromise ongoing clinical development programs and/or regulatory interactions.
  • The patient will receive treatment in a country or jurisdiction where Orca Bio’s plan is to seek regulatory approval to market the product.

Instructions for Making a Request

The request must be in writing, unsolicited and made by the patient’s treating physician. Please make sure that no patient identifying information is included. The request can be sent to expandedaccess@orcabio.com

Orca Bio will acknowledge receipt of all requests within three business days and will consider and determine whether to offer investigational product under the compassionate use program. There is no guarantee that therapy will be available to a patient at any given time. If approved, the patient must provide informed consent to receive the therapy and both the patient, and treating physician, will need to consent to comply with the safety and monitoring requirements defined by Orca Bio.

Orca Bio reserves the right to cease this expanded access program at any time if the product does not demonstrate a positive risk benefit to patients.

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