Company Directory

Single-Patient EA Policies/Criteria

MacroGenics designs its clinical studies for development of new potential therapies to determine the safety and efficacy for medical use consistent with approval requirements of the US FDA and similar regulatory authorities in other countries. Treating physicians should consider approved therapies for a patient’s disease as well as on-going clinical studies before seeking expanded access use with an investigational agent.

However, MacroGenics may, on a case-by-case basis, consider requests by treating physicians to file an investigational new drug application for expanded access to MacroGenics investigational products in Phase 2 or Phase 3 of development and for MacroGenics to supply such investigational product. There is no guarantee of expanded access to an investigational product. 

Available Therapies via Single-Patient EA

At this time, MacroGenics is unable to make investigational products available outside a clinical trial.

Single-Patient EA Policies/Criteria

Patients must satisfy the following criteria to be enrolled in the expanded access protocol:
Main Inclusion Criteria:
1. Molecular confirmation of a pathogenic or likely pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required.
2. Male or female patients aged ≥ 2 years.
3. Informed Consent signed by the patient, patient’s parent(s) or LAR indicating that they understand the purpose of and procedures required for expanded access and are willing to participate in the expanded access protocol.
4. Age-appropriate assent, if indicated.
5. In the opinion of the Investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses.
6. Able and willing to take investigational product (oral suspension) with food 3 times daily (TID). Ganaxolone must be administered with food.

Exclusion Criteria:
1. Pregnant or breastfeeding.
2. Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (magnetic resonance imaging).
3. Known allergic reaction or sensitivity to GNX or excipients.
4. Previously or actively participating in a clinical trial of GNX.
5. Participating in any study involving administration of an investigational agent, excluding devices.

Single-Patient EA Policies/Criteria

MEDRx USA, Inc. (MEDRx) is a clinical-stage pharmaceutical company that focuses on research and development of proprietary investigational products.

Investigational drug products generally can only be provided to subjects enrolled in clinical studies as such products are not approved by the U.S. Food and Drug Administration (“FDA”) as safe and effective for their intended use. While investigational product manufacturers may provide investigational products to certain patients under FDA’s Expanded Access Program (sometimes called “compassionate use”), if the patients meet certain criteria required by FDA law and regulation, at this time, MEDRx will not offer expanded access to investigational products outside of enrollment in clinical trials and is currently not accepting expanded access requests. If you have questions or need more information about our programs, please feel free to contact MEDRx at info@medrxusa.com . MEDRx endeavors to respond within five business days.

In line with the 21st Century Cures Act, MEDRx may revise this policy at any time.

Single-Patient EA Policies/Criteria

Meissa Vaccines, Inc. (https://www.meissavaccines.com) is developing investigational vaccines for the prevention of respiratory diseases. Since the vaccines are designed for prophylactic not treatment use, Meissa has chosen not to offer Expanded Access to these vaccines and will not accept Expanded Access requests. Meissa’s investigational vaccines will only be provided through ongoing clinical trials. To learn more about available clinical trials, please visit https://www.meissavaccines.com, or visit https://clinicaltrials.gov and search using the company name. 

Meissa Vaccines, Inc. Expanded Access Policy, rev. August 2021. 

Single-Patient EA Policies/Criteria

Consistent with Melinta Therapeutics’s mission to discover, develop, and commercialize a continual stream of novel antibiotics, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals and promptly make our medicines available to patients.  At Melinta Therapeutics, we believe that wherever possible, the use of an investigational medicine for a patient as part of a clinical trial is preferable; however, when patients with an unmet medical need are unable to access our investigational products through a clinical trial and all other options have been exhausted, we may be able to provide access through our compassionate use or expanded access program.

The evaluation criteria to determine if Melinta Therapeutics can provide expanded access to a specific Melinta Therapeutics investigational agent outside of a clinical trial include all of the following:
•    The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
•    The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
•    A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
•    Access is compliant with local rules and laws;
•    Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
•    Adequate supply of the investigational drug is available.

All requests must be submitted by the patient’s treating physician and Melinta Therapeutics may require more detailed information in order to fully evaluate a request. The treating physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Treating physicians seeking expanded access, meeting the above-mentioned evaluation criteria, should submit their requests to medinfo@melinta.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 3 business days after receipt. Melinta Therapeutics is committed to carefully evaluating requests on a case-by-case basis and in a fair and equitable manner; however, Melinta Therapeutics cannot guarantee access to any specific investigational drug by any individual patient.

If you are a patient with questions concerning access to our investigational products, either through a clinical trial or through expanded access, please consult with your treating physician.

Single-Patient EA Policies/Criteria

Currently, Immunicum does not have any investigational cell therapy products
available for Expanded Access

Available Therapies via Single-Patient EA

Immunicum is currently unable to offer an Expanded Access

Single-Patient EA Policies/Criteria

Criteria for Evaluation

Merus considers many factors when evaluating a request for early access of an investigational medicine, such as (but not limited to) the strength of the currently available data, including clinical data together with patient condition and the benefit-risk profile, the impact of providing access to an investigational medicine on its clinical development program, including the ability to complete patient enrollment and other necessary aspects of the clinical trials, the phase of development, and probability and timing of regulatory approval.

A patient may be considered for early access to an investigational medicine based upon evaluation of the following minimum requirements (and other requirements may apply based on the circumstances):

*There must be adequate data supporting use of the investigational medication in the treatment of the patient’s disease.
*There are no adequate standard or investigational therapies available.
*Sufficient safety and efficacy data must exist for use of the medicine at the time of request.
*Sufficient information must be made available to make a benefit-risk analysis consistent with the establishment of an early access program.
*Sufficient clinical data is available to identify an appropriate patient-specific dose.
*A patient’s treating physician and Merus’ clinical team both believe there is the potential for the specific patient under consideration to reasonably expect benefit from the treatment.
*Adequate supply exists to support both the ongoing clinical trials and approved early access, until and if a product becomes commercially available.
*The patient is not eligible for or cannot participate in a Merus-sponsored study for the therapy being requested.
*Early access will not adversely impact the clinical development program or the regulatory approval process.
*The request must be made, unsolicited, by the patient’s treating physician. The physician must be appropriately licensed.
*The treating physician must also agree to comply with the safety and monitoring requirements.
*Where appropriate, demonstration of efficacy and safety may be required for continued treatment.
*Early access is permitted or approved in the country, region, and/or state where the investigational medicine will be administered.
*Any condition, in addition to those listed above, that in the opinion of Merus may preclude appropriate use of the investigational medicine, will be considered.
Given the unique nature of individual requests for early access to investigational medications, there is no guarantee a request will be fulfilled.

Merus may revise, suspend or terminate the early access program at any time.

All early access requires approval by the Chief Medical Officer and Merus management and decisions will be made based on scientific and other evidence available to Merus at the time of the request.

Available Therapies via Single-Patient EA

MCLA-128 is available for patients with solid tumors that harbor an NRG1 fusion

Disease/Category-Specific EA Policies/Criteria

https://nrg1.com/

Single-Patient EA Policies/Criteria

At this time, Metacrine believes access to its products should be limited to clinical trials and does not offer an expanded access program or accept expanded access requests.

Single-Patient EA Policies/Criteria

Met Vital, Inc. will consider providing pre-approval access to our investigational drug, outside of the clinical trial setting, when certain conditions are met. These conditions are as follows:
• The patient to be treated has a serious or immediately life-threatening illness and there is no satisfactory alternative therapy.
• The patient is not eligible for, or cannot access, any ongoing clinical trials.
• The potential benefit of the investigational medicine to the patient, outweighs the potential risk. This should be evaluated by your physician and discussed in detail with the patient.
• There is an adequate supply of the investigational drug, meaning that MetVital, Inc has surplus investigational drug over and above what is required for the ongoing clinical trial.
• Providing the investigational drug will not interfere with the clinical trial that could support a marketing approval.

Requests for our investigational drug must come from a patient’s treating physician. The requesting physician must be willing to work on designing a treatment protocol and obtain the appropriate regulatory and ethics committee approvals from their Institutional Review Board (IRB). MetVital, Inc will not be responsible for the physician gaining IRB approval. The physician must also comply with various regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting.

Available Therapies via Single-Patient EA

Anhydrous enol-oxaloacetate for newly diagnosed Glioblastoma multiforme

Disease/Category-Specific EA Policies/Criteria

http://metvital.com/

Available Therapies via Single-Patient EA

Currently, none.

Single-Patient EA Policies/Criteria

Minoryx is a clinical stage biotech company committed to the development of safe and effective novel therapies for severe and life-threatening orphan CNS diseases with high unmet medical needs. We are a science-driven company committed to putting patients first. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
Clinical trials and the subsequent timely generation of safety and efficacy data are the most effective way of ensuring review and decision making by Regulatory Authorities. This ultimately has resulted in access to new, safe and effective approved medicines for patients.
Expanded access refers to the use of an investigational therapy outside a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.

Disease/Category-Specific EA Policies/Criteria

FDA Orphan indication was granted for the company’s lead program, leriglitazone (MIN-102), a novel, selective peroxisome proliferator-activated receptor gamma (PPARγ) agonist, for the treatment of X-ALD. On 4 April 2018, Minoryx opened the IND for leriglitazone (company code MIN-102) with a double-blind, randomized, placebo-controlled study to assess the effect of leriglitazone on the clinical progression in male patients with Adrenomyeloneuropathy (AMN). Recently, the FDA granted Fast Track Status to leriglitazone for development in patients with X-ALD.
An Early Access program for leriglitazone may be opened if Minoryx determines that all the following criteria are met:
• The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
• An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide Early Access to patients who do not have alternative treatment options
• Early Access does not impede or compromise the clinical development or regulatory approval of the drug under investigation
• There are sufficient clinical data available with respect to both the investigational drug and the disease condition for which the application is being sought, to anticipate that any potential benefits from treatment are likely to outweigh any potential risks to the patient
At this time, Minoryx believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapies. Minoryx continues to assess the eligibility requirements and criteria for Early Access to the investigational drug leriglitazone and will re-evaluate this policy from time to time.
If you have additional questions, please speak with your physician or contact clinical@minoryx.com. We anticipate acknowledging receipt of requests sent to this email within five business days.

Single-Patient EA Policies/Criteria

Minovia will make every attempt to respond to EAP request as soon as possible. An acknowledgement of receipt of request will be made within one week. Responses as to relevance of patient for EAP treatment may vary, based on the nature of the request and details of rationale for patient not being eligible for the currently open clinical trial.
Written requests from the patient’s healthcare provider should be submitted by email. All requests will remain confidential.

Disease/Category-Specific EA Policies/Criteria

Patients will be considered for EAP if they are not eligible for the currently open clinical trial of MNV-BM-BLD, carry a mitochondrial DNA deletion, and the mother is not a carrier of this deletion. Patient ineligibility for the open clinical trial may be due to their not meeting all inclusion/exclusion criteria (eg patient is below minimum age) or having a mitochondrial deletion syndrome other than Pearson Syndrome. 
Healthcare providers interested in information about the MNV-BM-BLD product for their patients can contact Minovia to request expanded access at expandedaccess@minovia.com

Single-Patient EA Policies/Criteria

Single-Patient Expanded Access Policies and Criteria - A licensed physician who believes their patient may benefit from access to a Mirati investigational medicine outside of a clinical trial should contact Early Access Care, by email Mirati.ExpandedAccess@earlyaccesscare.com or by contacting the Mirati Expanded Access Call Center 1-475-522-2200.

Available Therapies via Single-Patient EA

Expanded Access of Adagrasib (MRTX849) in Patients With Advanced Solid Tumors Who Have a KRAS G12C Mutation
Conditions: Advanced solid tumors harboring a KRAS G12C mutation
This is a multi-center, expanded access protocol to provide access to the investigational product, MRTX849, to adult patients with advanced solid tumors who have inadequate treatment options with available and approved therapies and who are otherwise ineligible for participating in clinical studies with MRTX829.

Disease/Category-Specific EA Policies/Criteria

https://www.earlyaccesscare.com/companies/mirati-therapeutics/adagrasib

Single-Patient EA Policies/Criteria

Moderna is working to deliver on the promise of mRNA science to create a new class of medicines for patients. As we advance our pipeline, patients, caregivers and healthcare providers may seek access to our investigational medicines outside of clinical trials and prior to regulatory approval. We understand the need for new therapies and the urgency behind requests for pre-approval access*.

While we continue evaluating the safety and effectiveness of this potential new class of medicines in clinical trials, we are not providing access to our investigational mRNA therapies or vaccines outside of these studies. We believe that participation in a clinical trial is the most appropriate way for patients to access any of our investigational treatments. We also believe that participation in clinical trials is the most efficient path to providing access to the greatest number of patients in need of a new therapy. Should our policy change over time, we will post updates on this site.

You can find links to information about our ongoing clinical trials on our website or go directly to www.clinicaltrials.gov and enter “Moderna” into the “other terms” search box. Please speak with your physician if you have any additional questions about our clinical trials or this policy. Our team plans to acknowledge receipt of requests sent to this email address from patients, families and healthcare providers within 5 business days.

*Includes requests for access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, as well as ‘Right to Try’ legislation (Public law no. 115-176).

Single-Patient EA Policies/Criteria

Molecular Partners AG is a clinical-stage biotech company developing DARPin® therapeutics, a new class of custom-built protein drugs designed to address challenges current modalities cannot. Molecular Partners develops DARPin® therapeutics for infectious disease, oncology including hematological malignancies, and ophthalmology. Ensovibep is currently being investigated in clinical trials for patients suffering from COVID-19 infection.

We at Molecular Partners, have a patient-focused approach in our development of medicines and we need to conduct clinical trials in order to gain regulatory approval, making ensovibep available to patients as quickly as possible. Molecular Partners understand the interest of patients in accessing ensovibep outside of clinical trials and prior to regulatory approval for the treatment of COVID-19. However, with alternatives which have achieved market approval or Emergency Use Authorization by the FDA these are more appropriate alternatives at current and we do not currently offer Expanded Access programs for ensovibep.

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

Information about our clinical trials, including eligibility criteria and locations, is available at www.clinicaltrials.gov.

Single-Patient EA Policies/Criteria

Momenta is committed to developing safe and effective medicines to treat rare immune-mediated diseases with high unmet medical needs. Our goal is to ensure access to our investigational drug, at the appropriate time and in a clinically appropriate manner for patients.

Momenta’s Early/Expanded Access Policy (EAP) refers to the use of an investigational drug outside of a clinical trial. This is initiated when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient, which is different from a clinical trial where more comprehensive safety and efficacy data are collected. At Momenta, we recognize and understand the need for an early/expanded access policy.

A number of factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies' guidelines should be taken into account when considering this kind of access.

Momenta’s Key Eligibility Requirements
To be eligible to access Momenta's investigational drugs under the EAP, participants must meet certain requirements, including, but not limited to:
- The illness must be serious or life-threating, with no other satisfactory treatment options (such as approved products or enrolling clinical trials)
- There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on all available safety and efficacy information
- Providing the investigational drug will not interfere with clinical trials that could support the investigational drug's development or marketing approval for the treatment indication

At this time, Momenta believes that participation in one of our clinical trials is the most appropriate way to access our investigational drugs. We do not currently provide our investigational drugs for use through expanded access.

Single-Patient EA Policies/Criteria

If you are a patient and interested in obtaining access to an investigational medicinal product, you should discuss available options with your treating physician. If a MorphoSys clinical trial may be a good treatment option for you, we recommend that your treating physician contacts us on your behalf via the contact information provided in ClinicalTrials.gov (search for Tafasitamab).

If you are not eligible for a clinical trial that is currently active, or if no appropriate clinical trials exist, please contact your treating physician to determine if an EAP may be the best or only treatment option for you. Your physician will find details on the EAP at ClinicalTrials.gov with the identifier NCT04300803.

Treating physicians should use the following contact (tafasitamab@clinigengroup.com) to receive the necessary request form, eligibility criteria and instructions for the EAP process. Receipt of requests will be acknowledged within two business days of receipt, approval or denial will usually be finalized within 2-3 business days. Before treatment can be started, the treating physician has to obtain IRB review.

MorphoSys is committed to a fair and impartial evaluation of each request for access to our EAP. Therefore, all decisions are based solely on clinical evidence and guided by the principles outlined below and by eligibility criteria for the EAP. In addition, MorphoSys intends to work in cooperation with local health authorities for the provision of an EAP.

Available Therapies via Single-Patient EA

Tafasitamab

Tafasitamab is used in combination with lenalidomide for the treatment of adult patients with relapse or refractory diffuse large B cell lymphoma (R/R DLBCL)

Single-Patient EA Policies/Criteria

Myeloid has one product, MT-101, in clinical trials. The trial is a Phase 1 multi-dose ascending trial for safety and tolerability. There is no expanded access to MT-101 until more therapeutic data is obtained.

Disease/Category-Specific EA Policies/Criteria

https://www.myeloidtx.com/products