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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

UCB

Phone Number & Email
Expected Application Timeframe
N/A
Epilepsy

This Compassionate Use Program (CUP) is setup to provide study patients with continuous access to Brivaracetam (BRV) for the time period between closures of the long-term follow up studies N01125, N01199, N01372, N01379, and N01315 and when BRV is commercially available.

Crohn's Disease

The objective of this program is to allow treating physicians to supply/continue to supply Certolizumab Pegol (CIMZIA®, CZP) to adults suffering from Crohn's Disease (CD), and who are considered not suitable for treatment, intolerant, have medical contraindications or had insufficient response with an authorized conventional therapy, including other authorized biologics.

Rheumatoid Arthritis

The objective of this Named Patient Program (NPP) is to provide continued availability of Certolizumab Pegol (CZP) to adult Rheumatoid Arthritis (RA) patients who participated in the open label studies C87015 (CDP870 015), C87028 (CDP870-028) and C87051 (CDP870-051). Physicians may use the option to continue offering patients CZP treatment or to transition patients off CZP to a standard care regimen.

Ultragenyx

Phone Number & Email
Expected Application Timeframe
N/A
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency
Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)
Mitochondrial Trifunctional Protein Deficiency
Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency
Glycogen Storage Disorders
Pyruvate Carboxylase Deficiency Disease
ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of
Barth Syndrome

This is a compassionate use study to allow patients already taking triheptanoin (C7) through previous studies to continue to receive the supplement. It will also allow triheptanoin supplementation in patients with qualifying disorders if they are failing conventional therapy.

X-linked Hypophosphatemia
Tumor-Induced Osteomalacia

Individual patient expanded access requests may be considered for patients who have no other treatment options

MPS VII
Mucopolysaccharidosis VII
Sly Syndrome

Individual patient expanded access requests may be considered for patients who have no other treatment options

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.

Single-Patient EA Policies/Criteria

Requests for early access to investigational therapies must be made by a qualified physician.

Usona Institute

Phone Number & Email
Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria

Licensed physicians should submit expanded access requests to Usona at EA@usonainstitute.org using the Usona Expanded Access Request Form. Requests for Expanded Access will be acknowledged within 5 business days of receipt. All Expanded Access use requests will be decided on a case-by-case basis at the sole discretion of Usona. All requests received will be reviewed anonymously by an internal Usona Committee to ensure patient safety and equality. The licensed treating physician must obtain, where applicable, all Ethics Review Board (ERB) or Institutional Review Board (IRB) approvals along with approval from the overseeing regulatory authority (e.g. FDA, EMA, Health Canada, etc.) prior to submission.

Expanded Access requests to Usona must meet ALL of the following criteria in order to be considered:
• The disease or condition for which use is requested is serious or life-threatening;
• The patient is ineligible or not able to participate in a clinical trial for the requested use and all approved treatment options have been exhausted without success and no satisfactory alternative treatment is available as determined by the requesting licensed physician;
• The requesting physician is a licensed physician and is authorized to deliver treatment as outlined in the request;
• There is sufficient clinical evidence to inform the safe use of the investigational drug under the requested use (at the requested dose and frequency of treatment);
• There is sufficient clinical evidence to suggest the requested use is expected to provide a potential clinical benefit to the patient (at the requested dose and frequency of treatment);
• The requested use would not negatively impact or interfere with active clinical trials or drug development programs of the applicable investigational drug;
• The treating physician has received approval by their overseeing regulatory authority (e.g. FDA, EMA, etc.) and where applicable, any Ethics Review Board (ERB) or Institutional Review Board (IRB) approvals
• The requested use complies with all applicable laws, rules and regulations;
• The requested use complies with Usona’s policies, including strict adherence to the ‘Usona Expanded Access Set and Setting Manual’;
• There is an adequate supply of the requested investigational drug available for the requested use.

Please note that Usona is only able to respond to Expanded Access requests from licensed physicians. Additional requirements apply to Expanded Access use of controlled substances, including specific personnel and facility requirements for expanded access use of drugs of the psychedelic drug class.

Available Therapies via Single-Patient EA

Psilocybin capsules (25 mg)

Disease/Category-Specific EA Policies/Criteria

https://www.usonainstitute.org/expandedaccess/