Company Directory

Single-Patient EA Policies/Criteria

Oblato Inc. will consider individual single patient access to OKN-007 in the United States for patients with Diffuse Intrinsic Pontine Glioma (DIPG), who cannot be treated by currently available drugs or clinical trials.

The primary option for patients to get access to investigational drugs is to participate in a clinical trial. Patients and physicians can learn more by reviewing information on current or planned clinical trials that is maintained by the U.S. National Institutes of Health.

We recognize, though, that there are extraordinary circumstances where access to these investigational drugs outside of a clinical trial is appropriate. Under our Access to Investigational Drugs Policy, a patient’s physician can request consideration for access if five basic criteria below are met:

- Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- Patient enrollment in a clinical trial is not possible.
- Potential patient benefit justifies the potential risks of treatment.
- Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

Available Therapies via Single-Patient EA

OKN-007 for patients with Diffuse Intrinsic Pontine Glioma (DIPG)

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

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

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

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