Company Directory

Single-Patient EA Policies/Criteria

At this time, based on the evidence generated to date, Pacylex has decided not to offer Expanded Access (EA) to any of our investigational products and will not approve EA requests. We will re-evaluate the status of our policy based on data generated from ongoing and future clinical trials.
This will be re-evaluated based on the criteria listed below.

3.1 Criteria
Where there is a possible need for patients to gain early access to a new medicine that is not generally available for prescription, Pacylex will consider the need for early access using the following criteria:

• Where it is expected that there is a need for ongoing treatment with one of our new medicines, we recognize that this must be through normal health services once the new medicine is available for prescription. As such we will only conduct clinical studies and provide early access outside of studies, in those countries where we intend to make the medicine available through normal prescription channels after its approval.
• We recognize that we must have a minimum level of evidence that the new medicine is likely to work before considering early access and must not provide excessive risk to any patients who may receive the new medicine early.
• While addressing this patient need, we will ensure that if we agree to provide early access to our new medicines, that there is sufficient medicine available to ensure the supply will not run out for those patients receiving it early, or for those patients in our clinical studies. This is because we want to ensure that when providing early access, it does not prevent us completing clinical studies and regulatory approval that would lead to wider access through normal routes of prescription.
• Any early access programs we provide will be in line with the laws and requirements of the country involved.

3.2 Continued treatment after clinical studies
To be sure that our new medicines will work effectively and be safe enough to be prescribed for the diseases patients are suffering from, we study them rigorously to see whether they provide adequate relief. The results of these clinical studies allow the regulatory authorities to independently assess whether to allow the new medicines to be prescribed. As this can take several years, patients who participate in our clinical studies may have to wait for a new medicine to become available on prescription.
Where there may be a benefit for a patient to continue to be treated after the clinical study has ended, but before it is available on prescription, we will decide if we have enough information to justify early access. This will depend on the severity of the disease we are studying, how much evidence we have that the medicine works as expected and is sufficiently safe, and whether alternative effective medicines are already available on prescription. If we plan for continued treatment after a clinical study, we will include this information in our study documents so that it is clear to each patient whether this is a possibility before they start in the study.
We understand that it can be difficult to decide to participate in a clinical study, so will ensure we make this information as clear as possible. To be sure we can assess whether the new medicine continues to work effectively and is safe enough to continue to be used during early access, we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine.

3.3 Compassionate or emergency use outside of clinical studies
The best way to see if a new medicine is sufficiently safe and effective is to run clinical studies. As such we prefer that, where possible, patients can participate in our clinical studies. We recognize however that not everyone has this opportunity. Where patients who cannot enroll in a clinical study are suffering from serious or immediately life-threatening diseases, and there are no satisfactory alternative treatments available, we will consider early access if there is enough evidence available in their condition (as described above) and whether there is a possibility of them being able to access the medicine on prescription in the future.
In the same way as after a clinical study, we want to be sure we can assess whether the new medicine continues to work and is safe enough to continue to be used. As such we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine.

3.4 Discontinuation of early access
When a new medicine receives approval from the regulatory authorities for the disease we are studying, and is available for prescription in the patient’s country, we will phase out the early access to allow the health system to prescribe the new medicine in the normal manner.
If, however, the medicine is approved but not available for prescription for some reason, and there is no alternative medicine available, we will work with country authorities to discuss if we can jointly set up a patient-assistance program to allow patients who are benefitting from it, who do not have alternative treatment options, to continue to receive the new medicine. Patient safety is our primary concern, and we respect patient safety by continually evaluating all the evidence that we have available to ensure that early access use of new medicines remains effective and appropriately safe. If, however, the evidence demonstrates that the new medicine is not going to be as sufficiently effective or safe as we had hoped, we will discontinue all early access and work with patients and their physicians to help them safely transition patients to other treatments. When regulatory authorities do not approve the use of the new medicine for prescription, because it is considered insufficiently safe or effective, and Pacylex discontinues its work to achieve regulatory approval, we will work with patients and their physicians to help them safely transition patients to other treatments.

Available Therapies via Single-Patient EA

PCLX-001 - Diffuse Large B-Cell Lymphoma (DLBCL) and Acute Myeloid Leukemia (AML)

Single-Patient EA Policies/Criteria

N/A – EA unavailable at this time

Available Therapies via Single-Patient EA

N/A – EA unavailable at this time

Single-Patient EA Policies/Criteria

We are unable to offer single patient expanded access at this time.

http://www.parion.com/pipeline/clinical-trials/

Single-Patient EA Policies/Criteria

PeproMene Bio Inc. is committed to developing safe and effective immune therapies such as BAFFR CAR-T cells to improve the lives of patients with cancer and immune disorders.

At this time, PeproMene does not offer an Expanded Access program. Our lead product, PMB-CT01 (BAFFR-CAR T cells) is investigational, which means that it has not been approved by regulatory health authorities, such as the United States Food and Drug Administration (FDA). While we fully understand that there may be desire to access to our products and recognize the significance of Expanded Access programs, our current priority is to advance our clinical trials as efficiently as possible to ultimately benefit the greatest number of patients. Therefore, PeproMene believes that participation in one of our clinical trials is the best way to access our investigational BAFFR-CAR T therapy. We encourage patients to speak with their physicians regarding participating in the clinical trials. Information on PeproMene’s ongoing clinical trials (NCT05370430 and NCT04690595) can be found in www.clinicaltrials.gov.

As more clinical data on the safety and efficacy of our investigational drugs become available, PeproMene will review and update its policy on Expanded Access. If you have questions about this policy, please contact info@pepromenebio.com.

Single-Patient EA Policies/Criteria

www.pfizercares.com

Single-Patient EA Policies/Criteria

ACCESS TO PHARNEXT INVESTIGATIONAL MEDICINES PRIOR TO REGULATORY APPROVAL
(FOR US PATIENTS ONLY)
Pharnext develops innovative new medicines designed to address unmet medical needs of patients in major therapeutic areas. We are focused on creating new medicines that address important, real world concerns of patients and their families. We currently have investigational treatments in clinical trials for a number of different diseases, as described in the PRODUCTS section of our website at https://www.pharnext.com/en/products/r-d-pipeline
Pharnext’s goal is to efficiently bring safe and effective medicines to all appropriate patients by conducting rigorous clinical trials and obtaining marketing approval by regulatory authorities, including the U.S. Food and Drug Administration (FDA). Prior to such approval, participation in one of our clinical trials is the optimal way for patients to gain access to our investigational medicines. However, we understand that some patients may not be able to participate in a clinical trial and may seek access to investigational treatments before they are approved. These situations are often referred to in various ways, including expanded access, early access, pre-approval access, compassionate use and emergency use.
If early data from clinical trials suggest that an investigational treatment can offer benefits for patients facing serious or life-threatening conditions, Pharnext may consider requests to provide access outside of a clinical trial. Our top priority when evaluating requests for expanded access to investigational medicines is to ensure that we are not putting patients at risk of unnecessary harm.
Pharnext will consider an expanded access program, or a single request for expanded access of an investigational medicine, only if all of the following criteria are met:
• The patient’s licensed physician submits an unsolicited request for expanded access
• The patient to be treated has a serious or immediately life-threatening illness and there is no satisfactory alternative therapy
• There are no comparable or satisfactory alternative therapies approved and available to treat the disease or condition
• There is preliminary clinical evidence of effectiveness and acceptable safety of the investigational treatment for that patient’s condition
• The potential benefit of the investigational medicine to the patients is greater than the potential risk
• Staff and other required resources are available to provide the product and there is an adequate supply of the investigational medicine
• Providing the investigational medicine will not interfere with the timely completion of clinical trials that could lead to marketing approval and thereby could delay its availability to other patients
• Expanded access will only occur in a country where Pharnext expects to file for regulatory approval of the product
Patients interested in obtaining access to a Pharnext investigational therapy must do so via their physician. Physicians should contact Pharnext Medical Information Center at medical@pharnext.com. All requests will be carefully considered, and a written or oral response will be provided to the treating physician within two weeks of receiving a request. For further information on available expanded access programs, visit www.clinicaltrials.gov and search ‘expanded access programs’.

Available Therapies via Single-Patient EA

NA

Disease/Category-Specific EA Policies/Criteria

NA - see above

Single-Patient EA Policies/Criteria

Pharvaris is committed to serving patients’ health needs by developing safe and effective therapies and making them available for use as quickly as possible. We believe that controlled clinical trials provide the best path for Pharvaris to generate the safety and efficacy information needed to obtain regulatory approval so that we can make our medicines available to all patients.

Pharvaris always seeks to provide the quickest and broadest access to its medicines, balancing benefit and risk to all patients. We support the principle of expanded-access programs consistent with the best standards for safe and effective use of an investigational therapy, as well as our ability to logistically provide a product. Pharvaris will consider requests for expanded access from qualified physicians, balancing multiple factors.

When evaluating potential use of an investigational therapy, we will consider several factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies’ guidelines including both patient and scientific perspectives. Eligibility requirements include but are not limited to:

• Sufficient scientific evidence to support both the safety and the efficacy of the compound’s mechanism for this indication
• A serious or life-threatening illness, with no other satisfactory treatment options (such as approved products or enrolling clinical trials)
• Sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on all available safety and efficacy information
• Support that providing the investigational drug will not interfere with clinical trials that could support the investigational drug’s development or regulatory approval for the treatment indication, which could affect future patient access
• The evaluation of these, and other, questions may require additional investigation or interaction with the requesting physician, other clinical investigators, or regulatory authorities. Favorable answers alone provide no guarantee that Pharvaris will be able to provide pre-approval access to an experimental medicine.

Requests for access to investigational therapies must be made by a qualified and licensed physician to Pharvaris (expandedaccess@pharvaris.com) and will be evaluated by a core team. Pharvaris anticipates it will acknowledge receipt of such requests within five business days after receipt of an inquiry.

Single-Patient EA Policies/Criteria

At Pieris Pharmaceuticals Inc., we are committed to developing novel medicines to improve the clinical outcomes of patients suffering from cancer and respiratory diseases. Cinrebafusp alfa (PRS-343) is in clinical development and currently being evaluated as a treatment for gastric cancer.

We understand the interest of patients in accessing cinrebafusp alfa outside of clinical trials and prior to regulatory approval. However, participation in one of our clinical trials is currently the most appropriate way to access our investigational therapies. Pieris does not offer cinrebafusp alfa via expanded access at this time.

We recognize the need for Expanded Access programs, and we will re-evaluate the status of our policy based on data from ongoing and future clinical trials.

If you have additional questions, please consult your physician or to learn more about our clinical programs, including eligibility criteria and locations to visit www.clinicaltrials.gov.

Single-Patient EA Policies/Criteria

There are seriously ill patients who will not be eligible for our clinical trials and may be in a high risk to experience an infection post a surgical intervention. In these circumstances, POLYPID will consider providing the requesting surgeon with pre-approved access to D-PLEX, for the prevention of infection of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:
• The patient is scheduled for an abdominal or a median sternotomy surgery for any reason;
• The patient is not eligible to participate in POLYPID clinical trials;
• The patient has a serious or life-threatening illness or condition and his/her likelihood to develop a serious infection due to surgical intervention is high;
• The patient has a disease or condition for which there is sufficient evidence of a projected benefit from the use of the D-PLEX and the benefit outweighs the known or anticipated risks;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting surgeon’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or a regulatory review or an approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available and can be supplied in the appropriate timeline of request.

Available Therapies via Single-Patient EA

D-PLEX, a new formulation of Doxycycline for local administration that is intended for prevention of Surgical Site Infections (SSIs) post cardiac surgery with median sternotomy and post abdominal surgery.

Disease/Category-Specific EA Policies/Criteria

https://www.polypid.com/wp-content/uploads/2020/08/PolyPid-Expanded-Access-Policy-FINAL.pdf

Single-Patient EA Policies/Criteria

Patient Eligibility Criteria:
To be eligible for access to an investigational product, a patient must meet the following criteria:

• Be an adult male with a confirmed diagnosis of X-linked adrenoleukodystrophy.
• Have undergone standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• Are not eligible or able to participate in any planned or ongoing clinical trial of the investigational product(s).
• Has manifestations of disease for which there is sufficient evidence of a projected benefit (e.g. based on prior positive clinical trial results) from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• Any other pertinent medical criteria for access to the investigational product, as established by Poxel.
• Investigational Product Criteria

In addition to the patient eligibility requirements, the investigational product must meet the following criteria:

• The product is in active clinical development.
• 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, and that there is sufficient investigational product available to ensure that supply constraints will not impede continued use of the product(s) by patient(s) or the conduct of our clinical trials.

Treating Physician Criteria and Responsibilities:
The physician(s) attending to the patient(s) who is/are receiving an investigational product through expanded use access is (are) properly licensed in the U.S. and fully qualified to administer the product. The physician must agree in writing to comply with:

• Any applicable legal and regulatory requirements related to providing an investigational product under Expanded Access.
• Any Poxel requirements in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property.

Available Therapies via Single-Patient EA

PXL065 for an adult male with a confirmed diagnosis of X-linked adrenoleukodystrophy.

Disease/Category-Specific EA Policies/Criteria

https://www.poxelpharma.com/en_us/pipeline/clinical-trials/expanded-access-policy

Single-Patient EA Policies/Criteria

At Prestige Biopharma, we are committed to developing safe and effective drugs to improve the health and well-being of patients. We recognize that some patients may have serious or life-threatening conditions and are not able to participate in our clinical trials. For these patients, Expanded Access to investigational drugs may be an option.

Currently, our investigational medicines are in early phase development, and we are not yet providing Expanded Access. We believe that participating in our clinical trials is the best way for patients to potentially access our investigational medicines prior to regulatory approval. We consider this approach to be the best opportunity to bring safe and effective treatments to patients. We encourage any person interested in gaining access to our investigational medicines to consult their physician regarding the possibility of participating in one of our clinical trials. Please review the information about Prestige Biopharma’s ongoing clinical trials found on this website as well as at https://clinicaltrials.gov/.

We wish to assure patients that we are constantly evaluating our policies and procedures. As such, Expanded Access may be reassessed in the future as our investigational medicines advance further in clinical trials and more data to support the safety and effectiveness becomes available.

If you have any questions about our Expanded Access policy or our clinical trials, please contact us at info@prestigebio.com.

Single-Patient EA Policies/Criteria

At PRG S&T, we commit to bringing new, innovative therapies to patients by conducting rigorous clinical trials and obtaining marketing approval by the FDA and other regulatory authorities. At this point of time, PRG S&T believes that participation in one of our clinical trials is the most appropriate way to access our investigational medicine. However, we may also provide our investigational medicine for use through expanded access program.

Requests for expanded access must be made by a physician on behalf of their patient. The eligibility for a patient to participate in the expanded access program will be reviewed and evaluated based on the patient’s medical information. The request for access to a PRG S&T investigational medicine will only be considered if the patient's treating physician is committed to, and supportive of, the requested treatment.

Questions with regards to the expanded access to PRG S&T investigational medicine may be sent to us via the contact information listed on our website. Should the circumstance permits change to our expanded assess policy, updates will be posted to this website.

Available Therapies via Single-Patient EA

Progerinin for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS) and Werner Syndrome (WS)

Single-Patient EA Policies/Criteria

The goal of our current clinical study program for PC945 is to enroll patients and obtain clinical data that is required by the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other regulatory authorities for review and approval of marketing applications for this investigational agent. We believe that focusing on clinical studies is the most appropriate way to achieve this goal; therefore, we do not currently offer access to PC945 outside of clinical studies and, at this time have no Expanded Access program (sometimes referred to as “compassionate use” or “pre-approval access”).