Company Directory

Single-Patient EA Policies/Criteria

Pafolacianine sodium injection (OTL38) can be accessed through On Target’s Expanded Access program by contacting On Target Laboratories directly. The patient would be required to travel to an approved clinical site, where the investigator has completed the necessary training to administer the drug and has access to the required Near-Infrared Camera device. This would include any clinical investigator who have been approved to participate and enrolled subjects in either the Phase 2 or Phase 3 Lung trials with the drug. The patient may qualify to be enrolled in an open study, if available, and On Target would not include this subject in the randomization plan and their data would not be included in the efficacy analysis of the open study. In the event this is not possible, an Expanded Access Submission would be required to be submitted under the open IND 118215 as a Protocol Amendment. On Target Laboratories would work with the selected clinical investigator to generate the submission, which would include the required information defined in CFR 312.305, including the rational for the intended use of the drug, criteria for the patient selection, and the benefit risk of utilizing the investigational drug and device. The patient selection criteria would match the inclusion criteria defined in the Lung Cancer Phase 3 trial.

Available Therapies via Single-Patient EA

Pafolacianine sodium injection (OTL38) for use as an adjunct imaging agent in the detection of lung cancer during surgery.

Single-Patient EA Policies/Criteria

Oncoceutics will consider single - patient expanded access for patients who meet the criteria for the company's existing expanded access program, as outlined on clinicaltrials.gov

Available Therapies via Single-Patient EA

ONC201 - H3 K27M mutant gliomas

Single-Patient EA Policies/Criteria

Expanded access is currently not available.

Single-Patient EA Policies/Criteria

At OncoNano Medicine, we are committed to developing products that bring new, innovative therapies, like pegsitacianine (ONM-100), to patients with serious or life-threatening illnesses or conditions. OncoNano is developing pegsitacianine to improve the ability of surgeons to identify cancerous tissue in the peritoneum during cytoreductive surgery.

At this time, OncoNano does not offer an expanded access program and is not accepting expanded access requests for investigational products, such as pegsitacianine.

OncoNano’s current focus and priority is to complete the product development program for its investigational product(s), such as pegsitacianine, in order to obtain the required safety and efficacy data needed for regulatory approval. We believe that focusing our resources on our clinical trial program is the best path forward to bring our investigational product(s) to patients as quickly and safely as possible.

As we continue to develop investigational product(s), we will review our expanded access policy for investigational product(s) and may make updates to this policy.

Patients can gain access to our investigational product(s), such as pegsitacianine, by participating in a clinical trial. If you or someone you know would like to learn more about OncoNano Medicine’s clinical trials, we encourage you to view our trials at www.clinicaltrials.gov.

If you have additional questions about OncoNano’s expanded access policy, please email us at info@onconanomed.com.

Single-Patient EA Policies/Criteria

To Whom It Concerns,
OncoVerity, Inc.’s (“OncoVerity” or “we”) mission is to reimagine a world where Cancer never wins. We
understand that success can only be achieved by collaborating closely with the families, patient groups,
clinicians, and regulators who share our mission of initially serving patients with AML.
Our focus is to advance Cusatuzumab toward regulatory approval and make it available to all who can
benefit from this therapy. Clinical research in the form of clinical trials (“Clinical Trials”) are key research
tools for advancing medical knowledge, utilizing innovative strategies to improve patient care. Clinicians
conduct these Clinical Trials in specific patient populations to determine whether an investigational
medicine works and is safe to use in patients. Participation in U.S. Food and Drug Administration (“FDA”)
or other relevant regulatory agency accepted Clinical Trials is the best way to access investigational
therapies. As such, at this time, participation in a Clinical Trial is the only way for patients to gain access
to the investigational therapy, Cusatuzumab, under development by Oncoverity.
We recognize that not all patients will meet the eligibility requirements for participation in a Clinical
Trial and as a result, understand that some may seek pre‐approval access when no other comparable
therapy is available for use, also referred to as expanded access, compassionate use, or named-patient
use (21 CFR part 312, subpart I).
Oncoverity relies on guidelines provided by the FDA and other regulatory agencies along with other
important factors when evaluating our ability to provide pre‐approval access for Cusatuzumab. Key
considerations include:
1) Whether there is sufficient evidence that the potential benefits to the patients outweigh the
potential risks, based on available safety and efficacy data
2) Whether sufficient clinical data is available to identify a recommended dose that might be
effective and is reasonably safe
3) Whether providing pre‐approval access outside of a Clinical Trial setting will compromise, delay,
or otherwise have a negative impact on ongoing Clinical Trials and/or future access by all eligible
patients
4) Whether there is adequate supply of product (Cusatuzumab) for Clinical Trials and any other
usage, such as expanded access
5) Whether it is feasible to provide any necessary follow up outside of the Clinical Trial setting
As we are about to embark on a Phase 2 study of Cusatuzumab, we are collecting additional safety and
efficacy data, which will be evaluated at the end of that study. Once that data is collected, we will reevaluate our policy on expanded access.
Here is the link to our open trial:
https://clinicaltrials.gov/study/NCT04023526?term=cusatuzumab&rank=1
At this time, we are not accepting requests for expanded access. If you have any additional questions
you may contact: helpdesk@oncoverity.com

Available Therapies via Single-Patient EA

Cusatuzumab currently being investigated in AML

Single-Patient EA Policies/Criteria

CUSP06 is currently in early development and OnCusp has not yet established a recommended dose and schedule for the treatment of patients outside of its clinical trial. Additionally, in order to assure an adequate supply of investigational medicine for its clinical studies, OnCusp will retain the ability to manufacture and supply investigational medicines in a fair and equitable manner and in a volume that provides a suitable supply for ongoing clinical trials and development programs. Hence, currently, OnCusp is unable to provide its investigational medicines on an Expanded Access or Right to Try basis.

Available Therapies via Single-Patient EA

OnCusp is currently not providing its investigational medicines on an Expanded Access or Right to Try basis.

Single-Patient EA Policies/Criteria

Named Patient Access (Single Patient IND in the U.S.) – where a qualified healthcare professional requests an investigational medicine for use for a single patient because, in their judgement, currently available therapies are not satisfactory. Provision of the investigational medicine by Ondine may or may not require approval by FDA as well as an Institutional Review Board.

Available Therapies via Single-Patient EA

Non-antibiotic nasal decolonization. See https://www.ondinebio.com/solutions/steriwave/ . This is a light-based therapy that can decolonize the nose of bacteria, viruses and fungi within 4 minutes. Typically used for pre-operative patients as well as for vulnerable populations or immunocompromised patients.

Single-Patient EA Policies/Criteria

Expanded Access Policy

All expanded access programs must be conducted in agreement with applicable legal and regulatory requirements related to providing an investigational product under expanded access protocols. Oneness Biotech believes that participation in one of our clinical trials is the best way to access our investigational drug product. We encourage patients to speak with their physicians regarding participating in clinical trials. In exceptional cases where patients with serious diseases are unable to participate in clinical trials and have exhausted all available options, requests for EAP will be considered, on a case-by-case basis, if it meets the following criteria listed below:

Criteria for Consideration of a Request for EAP

1. A patient has a serious, life-threatening or chronically debilitating disease;
2. There is a clear understanding that the investigational product is intended to treat diabetic foot ulcers for which use is requested by the licensed physician and the patient;
3. The investigational product is regarded by the licensed physician and the patient that the available clinical data presents evidence of efficacy such that a clinically meaningful benefit may be expected;
4. There is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition;
5. The patients are ineligible for, or otherwise unable to participate in, a clinical trial for the product;
6. The treating physician must complete FDA Form 3926 and is willing to open a Treatment IND with the FDA, including file paperwork with FDA and IRB (for many expanded access request types), and is responsible for patient care and reporting;
7. The treating physician should provide the completed Form to Oneness Biotech for review prior to submission to FDA;
8. The treating physician and the patient (and the patient’s family or custodians) agree to waive claims for damages against Oneness Biotech;
9. There is adequate supply of the investigational product to meet all needs for patients enrolled in ongoing clinical studies, and that designating investigational product for an expanded access treatment use will not compromise supply or otherwise postpone providing the new treatment, once approved, to the broader patient population;
10. Providing expanded access to an investigational product shall not negatively jeopardize the initiation, conduct, or completion of clinical investigation(s) and the overall development program to support registration of the product.

Available Therapies via Single-Patient EA

ON101 (under IND 079526) for indication of diabetic foot ulcers

Single-Patient EA Policies/Criteria

Opus Genetics 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 products, and to obtain regulatory approval.
Opus Genetics 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 Opus Genetics investigational products by contacting Opus Genetics.
Patients with serious or life-threatening diseases or conditions seek access to promising investigational medicines outside of a clinical study setting. The associated provision for requesting access to an investigational medicine is called ‘Expanded Access’ but is also known by other terms such as compassionate use” or “hospital exemptions”, “early access”, “emergency use”, “right to try access”, patients who seek access to investigational medicines outside of an established clinical study and prior to Health Authority marketing authorization may wish to do so because standard treatments have failed, they cannot tolerate already approved medicines, or because they are unable to participate in a clinical study.
The policy below sets forth the general guidelines that Opus Genetics will apply to its Expanded Access Program to ensure that requests for access to our investigational medicines are considered and processed in accordance with applicable legal and regulatory requirements, as well as in alignment with principles consistent with the Opus Genetics’ policies and procedures. The Policy is applicable globally and across all therapeutic areas. Further information about Opus Genetics’ clinical trials is available at Clinicaltrials.gov.
This Policy is designed to guide fair and equitable access decisions while maintaining Opus Genetics’ obligation to carry out the clinical development programs necessary to assess safety and efficacy of our investigational products. The guiding principles for granting access to individuals outside the clinical development programs build on our Guiding Principles for Expanded Access Programs and should consider:
•What actions by Opus Genetics will help ensure all patients are treated fairly and equally;
•Whether sufficient clinical data exists for a particular disease or condition to anticipate that the potential benefits outweigh the potential risks of the investigational medicine for that patient;
•Are the potential risks of treatment with the investigational drug reasonable in the context of the patient’s disease or condition being treated;
•Is there another investigational drug available with Opus Genetics or at another company that is more suitable;
•How will patients be selected for treatment;
•Whether a clinical trial participant who has had clinical success during enrollment in a clinical trial of investigative medicine will have continued access to the investigational medicine after completion of, or withdrawal from, the trial (i.e., bridging to commercial availability);
•Whether the granting of an individual request, given the current epidemiologic understanding of the number of patients who would have a similar clinical profile, would unduly burden the company with a magnitude of similar requests which should, by principles of justice and fairness, be provided to these other patients;
•How will patients be taken off treatment if the medication makes it to market or does not make it to market; and
•Whether it is feasible for the company to provide the required access for the duration of the patient’s necessity once any access is provided.
•Opus Genetics should evaluate, prior to the initiation of a program and periodically throughout a program, given the number of individual requests, whether a group or protocol-driven cohort program should be initiated.
1.1 Any use of a Opus Genetics investigational product outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including Opus Genetics policies and procedures.
1.2 In general, where permitted by local regulation, the investigational product supplied via Expanded Access may no longer be provided by Opus Genetics when it becomes available via the local healthcare system.
1.3 Opus Genetics may decide not to provide an investigational product under this policy if the Company does not intend to market the product in the country.
1.4 To qualify for expanded access under this procedure, the product must be under investigation in one or more clinical studies, have sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population, and assurance that the provision of the investigational product under this procedure will not interfere with or compromise the clinical development of the product.
1.5 Patients must be eligible for access to an investigational product, and must therefore meet the following criteria:
• Suffer from a serious or immediately life-threatening disease or condition;
• 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 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; and
• Any other pertinent medical criteria for access to the investigational product, as established by the Opus Genetics clinically or medically responsible individual.
1.6 This policy is posted on Reagan-Udall’s public website (Expanded Access Navigator) and apply to all Opus Genetics employees.

Lastly, Opus Genetics may consider closing a program for various reasons, including but not limited to:
•Commercial availability of the medicine for a particular need or condition, at which point it would be more broadly available to these patients in need;
•A definitive negative regulatory decision or a company decision to not proceed with clinical trials or commercialization for a particular disease or condition. In this instance, patients on expanded access treatment at the time of such a decision may continue to access therapy until disease progression;
•New information about the activity or safety of a medicine that could substantially change its benefit/risk profile; or
•Unexpected, limited product supply or other manufacturing issues.
 

Available Therapies via Single-Patient EA

Phentolamine opthalmic solution

Single-Patient EA Policies/Criteria

Currently, RadioMedix, Inc. and Orano Med, do not offer an expanded access program and are not accepting expanded access requests for AlphaMedix™. Our development resources are focused on conducting clinical trials that evaluate the safety and efficacy of our treatment.

Single-Patient EA Policies/Criteria

Orinove Inc. is committed to developing novel and selective medicines to patients with serious or life-threatening conditions. 
We are focused on enrolling and conducting the clinical trials necessary to gain regulatory approval to make our medicines available broadly to patients as quickly as possible. Orinove believes that participation in our clinical trial is the most appropriate way to access our investigational product therefore, at this time, Orinove is not making its investigational product available on expanded access.
We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy. In line with the 21st Century Cures Act, Orinove may revise this policy at any time. 
If you have questions about our investigational product or expanded access, please contact wangweiai1@orinove.com. Orinove anticipates that it will acknowledge receipt of any expanded access questions or requests within two weeks of receipt. 
For active clinical trials with Orinove’s investigational agents, please search “Orinove” at www.clinicaltrials.gov.

Single-Patient EA Policies/Criteria

In general, Orphazyme will consider granting access to an investigational drug on a case-by-case basis if an Early Access Program is open in the country of residence of the patient and the following criteria are met:
• The patient has a serious or life-threatening medical condition within the disease area and the patient population that Orphazyme is investigating and planning to pursue approval by the national regulatory authorities.
• The patient has no access to a suitable approved treatment for the disease condition or no adequate treatments are available.
• The patient does not qualify to participate in any ongoing or planned clinical trials in a reasonably accessible geographical location.
• The patient has a disease for which there is sufficient clinical evidence of benefit from the use of the investigational drug, and the benefits likely outweigh the known or anticipated risks.
• Providing Early Access does not in any way disrupt the clinical development program and/or the regulatory pathway towards marketing authorization leading to broader access.
• There is an adequate and sustainable supply of the investigational drug, beyond what is required for all ongoing and planned clinical trials.
• The request for Early Access is received from a licensed physician with expertise in treating the disease and understands the potential risks and benefits of the investigational drug.
• The requesting physician agrees to comply with Orphazyme’s Early Access Program specifications as well as with the applicable laws and regulations governing the use of the investigational drug. Early Access
ends once the investigational drug is commercially available or reimbursed in a country. If Orphazyme decides to discontinue clinical development of the investigational drug for a specific disease area, Early Access will be discontinued.

Available Therapies via Single-Patient EA

Arimoclomol for Niemann-Pick type C (NPC)

Disease/Category-Specific EA Policies/Criteria

Early Access Policy