Company Directory

Single-Patient EA Policies/Criteria

An Expanded Access Program provides HCPs and patients access to investigational therapies when the option of enrolling in a clinical trial may not be available. These programs may also be referred to as Compassionate Use Programs.

Currently, Gan & Lee is not offering an Expanded Access Program. To learn more about available clinical trials by Gan & Lee, please visit http://clinicaltrials.gov and search by company, disease, or compound.

Please feel free to contact us with any questions you may have.

Single-Patient EA Policies/Criteria

Gannex Pharma Co.,Ltd. (Gannex),  a wholly-owned company of Ascletis, is fully dedicated to the R&D and commercialization of drugs in the field of Non-alcoholic steatohepatitis（NASH）. Gannex conducts clinical trials in individuals to evaluate investigational medicines in order to obtain information on safety and efficacy that may be used to support marketing approval and subsequent wider accessibility of the product to patients. Investigational medicines are drugs that have not been approved by regulatory authorities.
Gannex has three clinical stage drug candidates against three different NASH targets Fatty Acid Synthase（FASN）, Thyroid Hormone Receptor-Beta（THR-β） and Farnesoid X Receptor（FXR）, and three pre-clinical stage NASH combination therapies. Gannex seeks to retain the ability to manufacture and supply investigational medicines in a fair and equitable manner and in a volume that assures adequate supply for ongoing clinical trials and development programs. Hence, at this time, Gannex is unable to provide its investigational medicines on an expanded access or right to try basis.  For patients seeking access to our investigational medicines before they are approved by a regulatory authority, participation in one of Gannex’s clinical trials is the most appropriate way to access these investigational medicines.  To learn more about available clinical trials by Gannex, please visit   www.gannexpharma.com, or visit https://clinicaltrials.gov and search by company, disease or medicine.

If you have additional questions, please speak with your physician or contact Gannex at ea@gannexpharma.com.
Consistent with the 21st Century Cures Act, Gannex may revise this policy at any time.

Disease/Category-Specific EA Policies/Criteria

http://www.gannexpharma.com

Single-Patient EA Policies/Criteria

https://www.gene.com/patients/clinical-trial-information/other-types-of-access#general-criteria

Single-Patient EA Policies/Criteria

GENSIGHT BIOLOGICS POLICY ON EXPANDED ACCESS TO INVESTIGATIONAL THERAPIES

Investigational therapy covered by this Policy

GS030 combination product for the treatment of Retinitis Pigmentosa

About Expanded Access

GenSight Biologics is focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Clinical trials form the foundation of our development programs. These trials are designed to assess the safety and efficacy of investigational medicines. Participation in a clinical trial represents the best way, in a controlled setting, to gain access to an investigational medication. Information about ongoing GenSightsponsored clinical trials can be accessed by consulting https://clinicaltrials.gov/. In some circumstances, a patient may not be able to participate in a clinical trial. Seeking use of an investigational medication under these circumstances is permitted by the FDA and commonly referred to as compassionate use or expanded access. Our policy is intended to comply with U.S. Food and Drug Administration (FDA) requirements for any such use or access.

At this time, given the early stage of development, we are not making our investigational therapy, GS030 combination product, available on an expanded access or compassionate use basis anywhere in the world. Generally, the safety and efficacy data needed to grant expanded access are not available until the initiation of phase 3 clinical trials. For now, participation in our phase 1/2 clinical trial, called PIONEER, is the only way to receive our investigational therapy.

If you are interested in participating in GenSight Biologics clinical trial and would like more information, or if you have any questions about this expanded access policy, please talk to your doctor or contact us https://www.gensight-biologics.com/form/contact-us/.

We will continue to evaluate the possibility of expanded access as we advance development of GS030 combination product. As authorised by the 21st Century Cures Act, GenSight Biologics may revise this posted expanded access policy at any time.

The availability of this policy or any revised version shall not serve as a guarantee of access to GS030 by any individual patient.

More information about GenSight Biologics’ ongoing clinical trial for Retinitis Pigmentosa, PIONEER, can be found by visiting the link: https://clinicaltrials.gov/ct2/show/NCT03326336.

12 October 2021

Available Therapies via Single-Patient EA

At this time, none of our therapies are available via single-patient EA.

Disease/Category-Specific EA Policies/Criteria

https://www.gensight-biologics.com/wp-content/uploads/2018/03/211012_GenSight-Biologics_Expanded-Access-Progam-Policy_GS030.pdf

Single-Patient EA Policies/Criteria

http://www.gsk.com/media/3368/compassionate-use.pdf

Go to GSK Compassionate Use Request Portal at https://gsk-cu-portal.idea-point.com/ to make a request.

Single-Patient EA Policies/Criteria

GMTUSA development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational device treatment, and to obtain regulatory approval.

GMTUSA is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to GMTUSA's investigational PCI-01 device treatment by contacting the Company.

The purpose of this policy is to describe the requirements for Expanded Access to GMTUSA investigational PCI-01 device treatment to patients outside of a clinical study.

Scope

This policy applies to provision of access to a GMTUSA investigational device product that is not approved for any purpose in the country from which the request is intended to be used. This also includes the time period between regulatory approval of an investigational product and its commercial availability in a country.

Policy Statements

Any use of a GMTUSA investigational product outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including COGMTUSA policies and procedures.

In general, where permitted by local regulation, the investigational product supplied via Expanded Access may no longer be provided by GMTUSA when it becomes available via the local healthcare system.

GMTUSA may decide not to provide an investigational product under this policy if the Company does not intend to market the product in the country.

A. Patient Eligibility Criteria

To be eligible for access to an investigational product, patients must meet the
following criteria:

• Suffer from a serious or immediately life-threatening disease or condition (COVID-19).
• Have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• Are ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
• The patient has a disease (COVID-19) for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• There is adequate information to support appropriate dosing for a special population patients such as pediatric, elderly, renal or hepatic disease, etc.
•Any other pertinent medical criteria for access to the investigational product, as established by the GMTUSA clinically or medically responsible individual.

B. Investigational Product Criteria

In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
• The product is under investigation in one or more clinical studies.
• There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
• The provision of the investigational product will not interfere with or compromise the clinical development of the product.

C. Treating Physician Criteria and Responsibilities

The physician(s) attending to the patient(s) who is/are receiving an investigational product through compassionate use access is (are) properly licensed and fully qualified to administer the product. The physician must agree in writing to comply with:
• Any applicable country-specific legal and regulatory requirements related to providing an investigational product under Expanded Access.
• Any CGMTUSA requirements in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property. A treating physician may submit questions or requests regarding expanded access to drfernandez@gmtpci.com

Single-Patient EA Policies/Criteria

Gyroscope Therapeutics’ mission is to preserve people’s sight and fight the devastating impact of blindness around the world. We embarked on this journey because each year millions of people lose their vision due to diseases that we believe may one day be treatable. Our current focus is to discover and develop pioneering gene therapies for one of the leading causes of vision loss – age-related macular degeneration (AMD).

To realise this mission, it is our duty to conduct the research and clinical trials necessary to evaluate the safety and effectiveness of our investigational medicines. We believe that rigorous clinical trials are the most appropriate way to do this. We are committed to working closely with researchers, doctors, clinical trial site teams, patients and families to enrol, conduct and analyse our studies. If successful, data from these studies may support submissions to regulatory authorities (such as the FDA and EMA) for potential approval – with the ultimate goal of making our medicines available to as many eligible patients in need as safely and quickly as possible.

At this time, we are not making our investigational gene therapy, GT005, available on an expanded access basis anywhere in the world (expanded access is sometimes also referred to as compassionate use or pre-approval access). For now, participation in one of our clinical trials is the only way to receive our investigational gene therapy. This decision was made after careful evaluation of many factors, including: the safety and effectiveness of GT005 is still being evaluated in early stage clinical trials; our manufacturing capacity for GT005; considerations related to one-time gene therapies; and, what we believe is in the best interest of patients.

We understand there are currently no approved treatments for dry AMD, which is why we are working hard to develop our gene therapies as quickly as we can. There are many clinical trials evaluating potential new therapies for this devastating condition. We recommend that you talk to your doctor about what is right for you.

If you are interested in participating in one of Gyroscope’s clinical trials and would like more information, or if you have any questions about this expanded access policy, please talk to your doctor or contact us. We anticipate acknowledging receipt of expanded access questions or requests sent to us within five (5) business days of receipt.

More information about Gyroscope’s ongoing clinical trials can be found by visiting the links below.

We will continue to evaluate the possibility of expanded access as we advance development of our investigational medicines. As authorised by the 21st Century Cures Act, Gyroscope may revise this posted expanded access policy at any time.

The availability of this policy or any revised version shall not serve as a guarantee of access to GT005 or any other investigational medicines by any individual patient.

Current trials of GT005 in geographic atrophy secondary to dry AMD:

FOCUS Phase I/II Trial

EXPLORE Phase II Trial

Available Therapies via Single-Patient EA

At this time, none of our therapies are available via single-patient EA. 

Disease/Category-Specific EA Policies/Criteria

https://www.gyroscopetx.com/expanded-access/