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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 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.

4 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


Phone Number & Email
Expected Application Timeframe
1 business day
Sphingomyelin Lipidosis

The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical trials. The program will provide access to olipudase alfa prior to registration and the availability of commercial product (including reimbursement where applicable) in the country of the patient.

Glycogen Storage Disease Type II
Glycogenosis 2

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this protocol is to provide enzyme replacement therapy with rhGAA on an expanded access basis, to severely affected patients with infantile-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored study currently enrolling patients with infantile-onset Pompe disease.

Autologous Stem Cell Transplant

The purpose of this program is to provide access to AMD3100 for patients who would benefit from an autologous stem cell transplant but who have either previously failed to collect enough cells for transplant following standard therapy or are not considered by their physician to have a reasonable chance of collecting enough cells for transplant.

Glycogen Storage Disease Type II (GSD-II)
Pompe Disease (Late-Onset)
Acid Maltase Deficiency Disease
Glycogenosis 2

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this expanded access study is to provide patients with Pompe disease in the United States (US), access to alglucosidase alfa produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration.

Pancreatic Cancer

The purpose of this study is to compare best supportive care plus oxaliplatin/ folinic acid/ 5-FU versus best supportive alone in patients with gemcitabine refractory pancreatic cancer.

Savara Inc.

Expected Application Timeframe
5 business days
Available Therapies via Single-Patient EA

Procedure for Submitting Requests to Savara:
Savara will consider expanded access requests from treating physicians subject to US laws and regulations. All requests should be submitted via e-mail to

Process for Review of Requests:
Savara is committed to a fair and impartial evaluation of each request for access to its investigational products. Therefore, all decisions are based solely on clinical circumstances and are guided by the criteria outlined below. Patients will be referred to ongoing clinical trials as the primary way to access investigational products. 

When evaluating requests for expanded access, Savara considers all of the following criteria: 
1. The patient for whom expanded access is requested suffers from a disease or condition that is serious or life-threatening. 
2. There are no comparable or satisfactory alternative therapies or clinical trials available. 
3. Sufficient preliminary efficacy and safety data exist to support an assessment that the benefit for the patient outweighs the potential risks and that the potential risks are not unreasonable in the context of the disease or condition being treated. 
4. Sufficient clinical data are available to identify an appropriate dose (amount and frequency) of the investigational drug. 
5. There is adequate drug supply to support the ongoing and necessary clinical trials as well as to support approved expanded access in a sustainable and equitable manner, until and if product becomes commercially available.
6. The patient is not eligible to participate in any ongoing clinical trials of the investigational drug. 
7. Expanded access will not adversely affect the clinical development program, in particular, the initiation, conduct, or completion of the clinical trials that are required for regulatory approval. 
8. The unsolicited request is made by a U.S. qualified and licensed physician who will take primary responsibility for supervising use of the investigational product from Savara and will comply with all applicable FDA regulatory requirements associated with treatment and use of an investigational product. 
9. All required regulatory and institutional approvals have been obtained. The patient must provide written informed consent. 

Requests for expanded access will be individually reviewed in accordance with these criteria. Savara is committed to evaluating all requests for expanded access in a fair and equitable manner. All requests will be evaluated by medical professionals and decisions will be based on available scientific evidence at the time of the request. 

Questions regarding Savara's ongoing U.S. expanded access program(s) can be forwarded to: 

This policy is not applicable to countries outside the U.S. If you have questions on expanded access in a non-US country, please submit your request to

Scilex Pharmaceuticals, Inc.

Single-Patient EA Policies/Criteria

Scilex Holding Company (Scilex) is dedicated to the development and commercialization of non-opioid pain management products using innovative delivery technologies and is actively evaluating the safety and effectiveness of these therapies.

Expanded Access Policy
The US Food and Drug Administration (FDA) has established criteria for the provision of investigational medicines* to patients outside of clinical trials (compassionate use/expanded access). That guidance provides that expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medicine for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available(1).
Expanded access may be appropriate when the following factors have been considered(2):

  • Patient has a serious or life-threatening illness or condition and is not responsive to or 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
  • Patient enrollment in a clinical trial is not possible
  • Potential patient benefit justifies the potential risks of treatment
  • Providing the investigational medicine will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational medicine for broader patient access
  • Adequate supply of the investigational medicine is available

Currently, Scilex is not accepting applications for expanded access of our investigational medicines. Our development resources are focused on conducting clinical trials that evaluate the safety and effectiveness of our investigational medicines for treating acute and chronic pain. Our clinical trial programs are the primary way to gain access to a Scilex investigational medicine and Scilex encourages patients to speak with their treating physicians about participating in a clinical trial whenever possible.
Scilex may reconsider making its investigational medicines available through an Expanded Access Program in the future as we continually evaluate each of our investigational medicines for appropriateness of expanded access based on FDA’s established criteria.
Healthcare providers, patients and caregivers interested in learning more about Scilex’s investigational therapies should contact Scilex at
Additional information regarding our clinical trials may be accessed on

*This policy is subject to change. Scilex may revise this policy at any time and may establish different expanded access policies for each investigational medicine under development. The posting of this policy by Scilex shall not serve as a guarantee of access to any specific investigational medicine by any individual patient.

**Investigational medicines have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medicine may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

(1) (accessed September 1, 2022); see also 21 C.F.R. 312.305(a)
(2) (accessed September 1, 2022)


Phone Number & Email
Expected Application Timeframe
24 hours
Single-Patient EA Policies/Criteria

SCYNEXIS is committed to making Investigational Products available to seriously ill patients who have exhausted other treatment options.  A treating physician, who is able to comply with the SCYNEXIS requirements, may request information about how to apply for access to SCYNEXIS’ Investigational Products by contacting the company.

Available Therapies via Single-Patient EA

SCY-078 is a triterpenoid, glucan synthase inhibitor, antifungal agent undergoing investigation for:
•    The treatment of invasive candidiasis, including candidemia (IC). 
•    The treatment of invasive aspergillosis (IA), including IA that is refractory to or intolerant of other antifungal therapies.
•    The treatment of invasive candidiasis in patients who are refractory to or intolerant of other antifungal therapies.

Servier Pharmaceuticals

Expected Application Timeframe
1 business day
Disease Attributes
Pathologic Processes
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue

This is an expanded access program to provide vorasidenib for treatment of patients 12 years or older with IDH1- or IDH2-mutated glioma.

Single-Patient EA Policies/Criteria
Single-Patient Expanded Access Policies and Criteria -. Physicians seeking general information may contact Servier by emailing .
Available Therapies via Single-Patient EA
Vorasidenib - The vorasidenib EAP is designed to provide access to vorasidenib for patients with IDH1- or IDH2-mutated glioma who are not eligible for other vorasidenib clinical trials, and who in the opinion of the treating oncologist would potentially benefit from treatment from vorasidenib.
Disease/Category-Specific EA Policies/Criteria

Shanghai SIMR Biotechnology Co., Ltd

Phone Number & Email
Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria
At SIMR we aspire to discover and develop differentiated therapeutics for the treatment of peripheral neuropathic pain. SIMR’s lead product, SR419, is a candidate drug with novel mechanism of action for the treatment of peripheral neuropathic pain, which is discovered and developed by SIMR. Peripheral neuropathic pain is the pain caused by damage or pathological changes of peripheral nerves, including post-herpetic neuralgia, diabetic peripheral neuropathy, chemotherapy-induced peripheral neuropathy, and postoperative peripheral neuropathy. The results of preclinical studies show that SR419 has excellent safety and pharmacokinetic properties. In addition, SR419 demonstrates the potential of better analgesic efficacy and fewer side effects than pregabalin, a commonly used first-line treatment for neuropathic pain. SR419 has completed phase I clinical trials in the Australia and China with positive safety results, and is planning to carry out international multi-center phase II clinical trials. At SIMR, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval, making SR419 available to patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access SR419 at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at SIMR understands the interest of patients in accessing SR419 outside of clinical trials and prior to regulatory approval for potentially life threatening diseases, however we do not currently offer any Expanded Access programs for SR419. SIMR recognizes the need for Expanded Access programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.
Disease/Category-Specific EA Policies/Criteria


Phone Number & Email
Expected Application Timeframe
3 business days
Hereditary Angioedema (HAE)

The expanded access program allows people to gain access to an unlicensed treatment on compassionate grounds. Lanadelumab, also known as TAK-743, is a medicine to help prevent angioedema attacks. This expanded access program enables these participants with a high unmet medical need to continue receiving lanadelumab during the interim period between completion of either the SHP643-301 (NCT04070326; SPRING study) or the TAK-743-3001 (NCT04444895) study and potential licensure of lanadelumab for the respective age group and/or treatment.

Gaucher Disease, Type 1

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this treatment protocol is to observe the safety of velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive (newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy (ERT) imiglucerase.

Silence Therapeutics plc

Single-Patient EA Policies/Criteria

At Silence, our vision is to transform peoples’ lives around the world by silencing diseases through our precision engineered medicines and driving positive change for the communities around us. As pioneers in the design and development of siRNAs (short interfering RNAs), we are advancing a new generation of medicines to potentially address the needs of patients who have limited or inadequate treatment options. Our investigational product candidates are designed to harness the body's natural mechanism of RNA interference (RNAi) to precisely target and silence disease-associated genes.

Silence conducts clinical trials to ensure that our investigational product candidates are safe and effective, which supports approval of the treatment by regulatory authorities and ultimately enables patients to access approved, safe and effective treatments.

At this time, Silence does not offer an expanded access program for its investigational products and these can only be accessed by participation in a clinical trial. If in the future Silence decides to offer an expanded access program, Silence will update its policy accordingly. If you are a patient who is interested in accessing one of our investigational products by participating in a clinical trial, please consult with your physician. Anyone who wishes to learn more about Silence’s investigational products or this policy can contact Silence by emailing More information on Silence’s clinical trials can be found at

Silk Road Therapies, Inc.

Phone Number & Email
Expected Application Timeframe
Available Therapies via Single-Patient EA

At this time Silk Road Therapies, Inc./Ipekyolu Ilac Ltd. is testing a topical pentoxifylline formulation under US IND, but exclusively conducted in a trial in Istanbul, Turkey. At this time, we are unable to import our topical formulation from Turkey and do not have sufficient supply to offer expanded access to this drug. Therefore, until such time as the proof of concept trial is completed, Silk Road Therapies, Inc. is not offering expanded access beyond participation in the Istanbul trial.

However, we anticipate completing the proof of concept trial 3Q2019 and having reformulated material available in the US 1Q2020. At that time, we will revise this expanded access policy and may permit access to our product for patients who are ineligible for entrance into our later phase III pivotal trial.

St. Jude Children's Research Hospital

Expected Application Timeframe
Single-Patient EA Policies/Criteria

Expanded access is used to describe treatment with an investigational product for a patient with a serious or life-threatening illness or condition when there are no comparable or satisfactory alternative treatments available and the patient is not eligible for a clinical trial. These investigational products are not approved by the U.S. Food and Drug Administration (FDA). The expanded access pathway is often used by commercial pharmaceutical companies to provide their products before they are approved.

St. Jude Children’s Research Hospital (St. Jude) is dedicated to its mission of advancing cures for pediatric catastrophic diseases through research and treatment. St. Jude provides the best possible care for our patients. We also make discoveries that will lead to better treatment for children and young adults around the world. We accomplish this by offering patients participation in clinical trials such as the LVXSCID-ND study (NCT01512888). 

St. Jude Children’s Research Hospital is not a commercial pharmaceutical company and does not have the capability to manufacture products at an industrial level. Supplies of the investigational product for the LVXSCID-ND study are limited and earmarked for the completion of a prospective research guided clinical trial. At this time, the safety and efficacy of the product is under investigation. St. Jude is not able to provide the investigational product in the LVXSCID-ND study for expanded access. If your child has been diagnosed with X-Linked Severe Combined Immunodeficiency (SCID-X1), please contact the physician below to discuss possible treatment options.

Disease/Category-Specific EA Policies/Criteria

Stanford University- Center for Cancer Cell Therapy

Single-Patient EA Policies/Criteria

We currently do not accept requests for expanded access use of our investigational products. Supporting single patient expanded access would negatively impact our resources and ability to complete the active clinical investigations.

We encourage anyone interested in receiving investigational products manufactured for Stanford Center for Cell Therapy investigator-initiated trials to contact the Principal Investigator through listing to see if they are eligible to enroll on an open study.

Steba Biotech S.A.

Available Therapies via Single-Patient EA

None are available via single-patient EA.

Disease/Category-Specific EA Policies/Criteria

Swedish Orphan Biovitrum AB (SOBI)