Expanded Access Policy

 Ridgeback Biotherapeutics, LP (“Ridgeback Bio”) is dedicated to working toward finding life-saving and life changing solutions for patients and diseases that need champions; Ridgeback Bio is the license-holder and manufacturer of monoclonal antibody, mAb114 – an investigational[1] therapeutic treatment for Ebola Virus Disease.[2]

Ridgeback Bio is committed to developing safe and effective therapies for rare diseases and providing those therapies to the broadest group of patients as quickly as possible. Inherent to that commitment is our support for expanded access and compassionate use programs when we have in hand substantial scientific evidence to support both the safety and the efficacy of an investigational product for an unique indication. In addition, provision of the investigational agent must also be logistically practicable.

An aspect of our commitment to patients and their families is Ridgeback Bio’s intent to make its eligible investigational therapies available to qualified patients worldwide under certain circumstances of critical and compelling need, when the data and other factors support this use. Early access (sometimes called “compassionate use” or “pre-approval access,” and referred to as “Expanded Access” in the United States) is a process that may help patients obtain investigational therapies that are not yet approved by the country’s regulatory agencies (e.g., the Food and Drug Administration (FDA)) outside of a clinical study.

Though Ridgeback Bio can make its investigational therapies available through early access, the preferred mechanism for access to any investigational therapy is by participation in a clinical study. We believe that investigational drugs should be studied in patients within clinical trials designed to obtain safety and efficacy data intended to support product approval and subsequent wider patient accessibility in the most efficient and expedient manner possible.

Clinical studies collect the information necessary to understand the safety and efficacy of the potential therapy allowing for regulatory authority review and potential approval. Ultimately, we believe that approval by regulatory authorities gives the opportunity for the broadest access because regulatory approval allows physicians to prescribe the therapy to any patients who might benefit. To participate in a clinical study, you/your child must meet certain eligibility criteria.

For information on ongoing clinical studies in which mAb114 is offered as the investigational treatment:

·       Visit ClinicalTrials.gov and search registered clinical studies for the term “mAb114”; or

·       Contact Ridgeback Bio directly via email to:  EAP-requests@ridgebackbio.com.

In addition, mAb114 is currently under study in the Democratic Republic of the Congo through an Expanded Access Protocol administered as part of the Monitored Emergency Use of Unregistered and Investigational Interventions for Ebola virus disease (MEURI).[3]

Requests for access to experimental therapies must be made by the patient’s qualified and licensed treating physician, unsolicited by Ridgeback Bio or any other individual or organization. Written requests should be directed to Ridgeback’s “EAPRequest” email address (specified above) and should include:

·       Physician contact information; and

·       Brief narrative of the case for which expanded access is being requested, including date of confirmatory diagnosis of Ebola Virus Disease (Zaire); and

·       The number of treatment courses being requested (in the event of more than one affected patient with confirmatory or suspected diagnoses).

Individual written requests for Expanded Access to mAb114 will be promptly referred to a core team for evaluation; Ridgeback Bio anticipates it will acknowledge receipt of such requests within no more than 48 hours of receipt of the request form.

Please noteCurrently, Ridgeback Bio’s primary aim is in satisfying demand for mAb114 investigational product in support of outbreak mitigation efforts and clinical study objectives in the Democratic Republic of Congo and surrounding territories.[4] For that reason, Ridgeback Bio must first establish that:

1       access to mAb114 through a written Expanded Access request will not compromise ongoing clinical trials or the regulatory pathway for approval of mAb114; and

2       that an adequate supply of the investigational product exists to attend to outbreak mitigation and clinical trial objectives; and

3       logistically, that the investigational product is feasible to make available and can be administered properly outside of a clinical trial setting. 

If these conditions are met, Ridgeback Bio will consider compassionate use requests from treating physicians subject to local/national laws and regulations and in consideration of national and global public health objectives.

Ridgeback Bio does not require patients nor requesting physicians to pay for the cost of investigational products. In addition, Ridgeback Bio is not responsible for the cost of patient care under the study protocol, unless a prospective written agreement is in place for Ridgeback Bio to reimburse or cover any specific costs associated with the use of Ridgeback Bio’s investigational product in this setting.

As authorized by the 21st Century Cures Act, Ridgeback Bio may revise this expanded access policy at any time. Additionally, the posting of this policy by Ridgeback Bio shall not serve as a guarantee of access to any specific investigational drug by any individual patient.

POL001 (1.0) Version Date:  20 September 2019

[1] An investigational therapy is a potential new therapy that has not yet been approved by regulatory authorities such as the Food and Drug Administration (FDA) in the United States or the European Medicines Agency (EMA) in Europe.

[2] Under the 21 Century Cures Act, the manufacturer or distributor of one or more investigational drugs for the diagnosis, monitoring or treatment of one or more serious diseases or conditions shall make available its policy on how it evaluates and responds to requests submitted under Section 561(b) of the Federal Food, Drug and Cosmetic Act (FD&C Act) for provision of such a drug.

[3] https://www.who.int/emergencies/ebola/MEURI-Ebola.pdf

[4] WHO Director General declared the Ebola Virus Disease outbreak in Democratic Republic of Congo a Public Health Emergency of International Concern on July 17, 2019. https://www.who.int/news-room/detail/17-07-2019-ebola-outbreak-in-the-democratic-republic-of-the-congo-declared-a-public-health-emergency-of-international-concern