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If you know the name of a drug manufacturer, you can easily search using the alphabetical menu below. Or you can search in free text – simply type the disease, drug, company name, or other detail into the Search bar below and click on the magnifying glass. For broadest results, type the terms without quotation marks; to narrow your search to an exact match, put your terms in quotation marks (i.e.; “breast cancer”). See Search Tips for tips and definitions to make your searches more effective.

The web links in this directory are public information provided to the Foundation and the acknowledgement times listed are the number of days or hours for a company to acknowledge receipt of request, not to make a decision. Data contained in this directory is provided either directly by the company or by ClinicalTrials.gov for the companies listed in this directory.

The Expanded Access listings provided on this website are limited to those companies currently in the Company Directory and include listings for expanded access which are Available, Temporarily Not Available and Approved for Marketing, which in some cases includes treatments which have not yet reached the market. In addition, many companies are willing to consider expanded access requests even if they do not show active Expanded Access Listings in this directory. Contact the company to explore the possibility of individual access to an investigational therapy.

To learn more about clinical trials and expanded access, please visit our patient and physician guides on this website.

A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
Company Name
Phone Number & Email
Company Acknowledgement

On Target Laboratories, Inc.

Phone Number & Email
Expected Application Timeframe
3-5 business days
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.

Oncoceutics, Inc.

Expected Application Timeframe
48 hours
Glioma

The objective of this expanded access program is to provide ONC201 to eligible patients with previously-treated glioma that exhibits the H3 K27M mutation and/or that is located in the midline region of the brain.

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

Oncopeptides, AB

Expected Application Timeframe
24 hours
Available Therapies via Single-Patient EA

Melphalan Flufenamide (melflufen)

An Expanded Access Program (EAP) Protocol for Melphalan Flufenamide (melflufen) in Combination with Dexamethasone in Patients with Triple Class Refractory Multiple Myeloma

EA Policies/Criteria

To provide early treatment access and evaluate the safety of melflufen and dexamethasone in patients with triple class refractory multiple myeloma prior to commercial availability of the medication.

Ondine Biomedical

Expected Application Timeframe
7 business days
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.

Orinove Inc.

Phone Number & Email
Expected Application Timeframe
2 weeks
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.

Orphazyme

Expected Application Timeframe
5 days
Niemann-Pick Disease, Type C

NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol. Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.

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

Otsuka America Pharmaceutical

Phone Number & Email
Expected Application Timeframe
1 week