Company Directory

Single-Patient EA Policies/Criteria

We do not plan to offer Expanded Access for our studies. 

Single-Patient EA Policies/Criteria

At Recursion Pharmaceuticals, we are committed to radically improving the lives of patients and their families by developing therapies that will make a lasting, positive, and transformative impact on diseases and conditions for which there are high unmet needs.

We believe that the best way to succeed in our mission is through well-designed clinical trials that determine the safety and effectiveness of investigational medicines. We understand that in some rare and specific
circumstances, when enrollment into a clinical trial is not possible, physicians caring for patients with serious or life-threatening conditions or diseases may seek special access to investigational medicines. Recursion will consider individual Expanded Access requests for an investigational medicine outside of an ongoing clinical trial for the indication being investigated by the company.
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A licensed treating physician must submit the request for Expanded Access in writing by contacting
ExpandedAccess@Recursion.com. The physician must be able and willing to obtain a treatment IND, or regional equivalent, and Institutional Review Board or Ethics Committee approval, for the patient prior to initiating treatment with our investigational medicine.

A team at Recursion will review the information provided and determine whether it is in the best interests of the patient and our development program to support the Expanded Access request. The evaluation will be based on patient eligibility, risk-benefit analysis, and impact to clinical development programs including drug supply. A response will be sent to the requesting physician communicating the approval or denial of the request for Expanded Access. We endeavor to respond to Expanded Access requests within one week after the steps for submission are completed.

Single-Patient EA Policies/Criteria

Consistent with RemeGen’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or 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, RemeGen will consider providing a requesting physician with pre-approval access to a specific RemeGen investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.
The following criteria are typically required before RemeGen would consider patients eligible for expanded access to a RemeGen investigational product. All criteria are subject to local laws and regulations.
• The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
• The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
• The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
• The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive which typically occurs when RemeGen has decided to seek marketing approval in at least one major market globally.
• RemeGen has decided to seek marketing approval in at least one major market globally.
• The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
• The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.
RemeGen cannot approve expanded access use requests in cases where doing so would violate company policy, local laws or regulations, or if access would present an unacceptable benefit/risk profile, as deemed by medical personnel of the company.
We commit to a careful and fair evaluation of each request by the appropriate medical experts at RemeGen within the boundaries of local laws and regulations. All requests for expanded access (typically made by the treating physician) will be acknowledged by medical personnel from RemeGen (or our agent) within 7 business days. Should the request be approved, the requestor must enter into an Expanded Access Agreement with RemeGen.

http://www.remegen.com/index.php?v=show&cid=113&id=1006

Available Therapies via Single-Patient EA

Telitacicept for patients with Generalized Myasthenia Gravis

Single-Patient EA Policies/Criteria

The following criteria are typically required before RemeGen would consider patients eligible for expanded access to a RemeGen investigational product. All criteria are subject to local laws and regulations.
• The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
• The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
• The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
• The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive which typically occurs when RemeGen has decided to seek marketing approval in at least one major market globally.
• RemeGen has decided to seek marketing approval in at least one major market globally.
• The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
• The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.

Available Therapies via Single-Patient EA

Telitacicept for patients with moderate or severe SLE

Single-Patient EA Policies/Criteria

Reneo is committed to developing promising new therapies to address the unmet medical needs of patients suffering from rare and seriously debilitating diseases. We currently have a late-stage clinical trial planned for the treatment of Primary Mitochondrial Myopathies (PMM) and early-stage studies for Glycogen Storage Disorder V (GSDV, also known as McArdle Disease) and Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD). Our goal is to provide access to our medicines at the appropriate time and in a manner that is most beneficial to the relevant patient population. We believe enrollment in our ongoing clinical trials is the safest and most effective way of achieving this goal. We do recognize that some patients will not be eligible for our clinical trials and may wish to access our products through expanded access. However, at this time, we do not have the resources available to offer expanded access use of our investigational medicines. We encourage all patients and physicians who are interested in accessing our investigational medicines to visit the clinical trial section below to find out about enrolling in clinical trials.

Available Therapies via Single-Patient EA

N/A

Single-Patient EA Policies/Criteria

Renibus Therapeutics will evaluate and respond to a request that it receives on a case-by-case basis.

Available Therapies via Single-Patient EA

RBT-9 for the treatment of COVID-19 at the early stage (Stage I) of infection in patients who are at high risk for disease progression.