Pharmaceutical Companies Using EAS

Migalastat (Galafold)

Migalastat HCl is currently licensed in the United States, Canada, Europe, Switzerland, Israel and other countries for the long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease with a mutation amenable to this treatment. The market authorization process is ongoing in multiple other countries.

Migalastat therapy is dosed as one capsule, 123 mg, every other day. Capsules are blister-packaged with an inactive reminder day to assist compliance.

Amicus will receive requests for access prior to market authoziation, through Early Access Care, for individuals meeting the following criteria:

• Confirmed GLA mutation predicted to be responsive to migalastat in the human embryonic kidney (HEK-293) cell-based assay
• 16-74 years of age
• Strong clinical indication for treatment of Fabry disease
• No other treatment option including either unsuitable for ERT or unable to access ERT
• Appropriate female and male contraception
• Willing to receive treatment with migalastat HCl via this program including having signed an authorization for sharing clinical data

The following are exclusion criteria, meaning that migalastat will not be approved in the following:

• Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute/m2
• Scheduled for renal or other organ transplant or replacement therapy
• Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta) or REPLAGAL™ (agalsidase alpha)
• Contraindication to migalastat, i.e., sensitivity to other iminosugar such as miglustat, miglitol
• Treated with another investigational drug within 30 days of start of migalastat HCl treatment
• Unable to comply with requirements or deemed otherwise unsuitable for study entry in the opinion of the requesting physician.

Serious Adverse Events (SAEs) - All SAEs must be reported to PhV.Migalastat_SO@quintiles.com within 24 hours of any knowledge of the event.

Non-Serious Adverse events (AE) and Product Complaints (PC) must be reported within 24 hours of any knowledge of the event to PhV.Migalastat_SO@quintiles.com

Pregnancy exposure, lactation exposures and other Special Circumstance events must be reported within 24 hours of any knowledge of the event to PhV.Migalastat_SO@quintiles.com

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Available in These Countries:

• Canada
• Chile
• Egypt
• India
• Namibia
• New Zealand
• Turkey
• Angola

ATB200 / AT2221 (cipaglucosidase alfa / miglustat)

ATB200 is a novel rhGAA currently being developed by Amicus Therpeutics, Inc. as a potential next-generation ERT for the treatment of adult patients with Pompe disease. Physician inquiries should be directed to Early Access Care at ATB200.NamedPatient@earlyaccesscare.com or 1-203-441-7938 to speak with one of our coordinators.

Serious Adverse Events (SAEs) must be faxed to +1 646-963-2056 within 24 hours of any site staff knowledge of the event. The form for reporting of SAEs is here.

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Available in These Countries:

• United States of America
• Croatia
• Germany
• Italy
• Portugal
• Taiwan

BHV-0223

Biohaven Pharmaceuticals has developed a novel sublingual formulation of riluzole, BHV-0223, for the treatment of ALS. BHV-0223 (sublingual) dissolves under the tongue in seconds and effectively delivers riluzole into the bloodstream through sublingual absorption. Clinical studies demonstrate that BHV-0223 (sublingual) requires a lower dose of riluzole (40mg) than the tablet (50mg) to achieve the same blood levels.¹

BHV-0223 is still an investigational drug. FDA approval and commercial availability are anticipated in mid- to late 2019. The company has opened an Expanded Access Program (EAP) in an effort to bring this investigational treatment option to People with ALS (PALS) today. [ClinicalTrials.gov (NCT03537807)]

BHV-0223 (sublingual)

Riluzole is the only FDA-approved treatment demonstrated to extend time to tracheostomy and survival in ALS.[Rilutek USPI] Reports of tolerability issues with swallowing riluzole may result in premature discontinuation or altering its delivery (e.g. crushing and taking with food or chewing), reducing the potential benefit of riluzole.²

BHV-0223 sublingual formulation of 40mg bypasses gastrointestinal absorption, avoids initial high concentration first pass liver metabolism and provides an ease of administration (no swallowing or liquids required) for patients with dysphagia.

Value of BHV-0223 Sublingual Early Access Program

The FDA works with pharmaceutical companies to allow patients access to investigational drugs outside of the clinical trial setting before approval through an early access program.

The BHV-0223 (sublingual) EAP offers an opportunity for PALS to try a sublingual formulation of riluzole at no cost to the patient or institution. It will allow an opportunity to collect serial clinical data from a broader ALS patient population, to evaluate patients' views on ease of administration, tolerability, safety and clinical benefits of BHV-0223. [ClinicalTrials.gov (NCT03537807)]

The protocol has been reviewed by the FDA and approved by an IRB. Click here for additional information about the BHV-0223 protocol.

Enrollment to the BHV-0223 Program is now closed.

Safety Reporting: A written description of any serious adverse event, using the PPD SAE report form, must be sent to PPD PVG by facsimile (fax) within 24 hours after awareness of the event: North America - 1-888-488-9697

If a form is unable to be submitted within 24 hours, the SAE may be reported by telephone via the Safety Hotline Number: North America - 1-800-201-8725

Webinar on BHV-0223 Expanded Access Program:

A brief webinar, hosted by the ALS Association, provides an overview of the Expanded Access Program. Dr. Richard Bedlack MD, PhD, Director of Duke ALS Clinic, Duke University provides insights into the Expanded Access Program. The webinar is available here.

1. Qureshi et al. A Phase 1 Study to Evaluate Bioequivalence Between BHV-0223 40mg Zydis Sublingual Formulation and Riluzole 50mg Oral Tablet in Healthy Volunteers. Poster presented at American Academy of Neuromuscular and Electrodiagnostic Medicine (AANEM) Clinical Conference; 2018 Oct 9-12, Washington D.C.
2. Introna et al. Adherence to ALS treatments. Neuropsychiatric Disease and Treatment 2018;14:193-203.

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Available in These Countries:

• United States of America

Viaskin Peanut (DBV712) EAP02

This is a treatment-use study that permits peanut-allergic individuals who participated in a DBV712 clinical study to be treated with DBV712 250 mg as open-label treatment. Additionally, participants treated with DBV712 for more than one year through compassionate use or similar regulatory-approved path may enroll and continue to be treated with DBV712 250 mg.

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Available in These Countries:

• United States of America
• Australia
• Canada
• Germany

Viaskin Peanut EAP01

Investigational Viaskin® Peanut therapeutic treatment is based on epicutaneous immunotherapy, or EPIT™. This potential new class of immunotherapey is designed to work by delivering allergens to the immune system through intact skin using our proprietary Viaskin® technology. View Additional Information on the Viaskin Platform.

Clinical Trials

The REALISE (Real Life Use and Safety of EPIT) Phase 3 clinical trial in children 4 to 11 years of age, has completed recruitment (NCT 02916446). For children completing the REALISE clinical trial, an Expanded Access Program is available for the continued treatment with Viaskin® Peanut.

Requesting Expanded Access

All requests for Expanded Access must be made by a treating physician on behalf of the child and the child's parent or adult guardian. DBV Technologies has opened an Expanded Access Program for Viaskin® Peanut post-trial treatment. A treating physician may submit questions or requests regarding Expanded Access by emailing dbvtechnologies.eap@earlyaccesscare.com or by calling Early Access Care at 203-441-7939 and speaking with an Early Access Coordinator. Requests will be acknowledged within one business day. No patient is guaranteed entry into the program solely by applying.

Physicians must follow local laws and regulations appropriate for the country originating the request.

Eligibility Criteria: In order to enroll in the Expanded Access Program, the physician must provide documentation of patient participation and completion in the REALISE study. Patients must provide informed consent for Expanded Access. Additionally, patients must be willing to continue following a strict peanut-free diet and carry an injectable epinephrine.

Safety Reporting: In addition to other obligations for safety reporting, as required by federal regulations, a written description of any serious adverse event (SAE), using the SAE Reporting Form, must be submitted to DBV Technologies within 24 hours after awareness of the event. Please submit SAE to: pharmacovigilance@dbv-technologies.com

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Available in These Countries:

• United States of America
• Canada

DKN-01

Leap Expanded Access Policy is available here.

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Available in These Countries:

• United States of America

Adagrasib (MRTX849)

Adagrasib (MRTX849) is a potent, highly selective and orally available small molecule inhibitor of a form of KRAS that harbors an oncogenic substitution mutation (G12C). KRAS G12C is a well-validated driver mutation present in approximately 14% of NSCLC adenocarcinoma patients and 5% of CRC patients.

Adagrasib has demonstrated broad-spectrum antitumor activity across a panel of KRAS G12C-positive patient- and cell-derived in vivo tumor models, including complete tumor regression in a subset of these models. MRTX849 is now in the clinic and has the potential to provide a long-awaited targeted therapy option for patients exhibiting a KRAS driver mutation.

Mirati has made adagrasib (MRTX849) available through and intermediate expanded access protocol (NCT 05162433) for patients ineligible for a clinical trial of adagrasib (MRTX849). Additional information is available here.

Criteria

Inclusion Criteria:

• Histologically or cytologically confirmed diagnosis of an advanced/metastatic solid tumor
• Confirmed presence of a KRASG12C mutation
• Ineligible for an ongoing clinical trial of MRTX849
• No available or not eligible for standard of care treatment
• Adequate organ function
• CNS Metastases (within set parameters) are allowed
• ECOG performance status of ≤ 2

Exclusion Criteria:

• History of intestinal disease, inflammatory bowel disease, major gastric surgery, or other gastrointestinal conditions likely to alter absorption of MRTX849 treatment or result in inability to swallow
• Prior therapy targeting a KRAS G12C mutation
• Other active cancer

Contact Information: A treating physician may submit questions or requests regarding Expanded Access by contacting the Mirati Call Center (475) 522-2200 or by emailing Mirati.ExpandedAccess@earlyaccesscare.com

Request Procedure: Patients interested in obtaining access MRTX849 must do so via their physician. A licensed physician who believes their patient may benefit from access to MRTX849 should contact Mirati through the contact information above, to make the request on behalf of the patient.

Anticipated Timing: Mirati will acknowledge receipt of any Expanded Access questions or request from licensed physicians within one (1) business day of receipt. Making a request does not guarantee the granting of access to an investigational drug by Mirati. If approved, the Expanded Access supply of investigational medicine may be continued until the drug is approved for use for the indication through the local healthcare system, or until, in the opinion of the treating physician, there is no treatment benefit. However, Mirati cannot guarantee drug supply. Mirati reserves the right to restrict access or discontinue the Expanded Access investigational drug at any time.

For information on Mirati clinical trials, please visit ClinicalTrials.gov or visit Mirati Clinical Trials.

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Available in These Countries:

• United States of America

Vorasidenib

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

Detailed Description: This is a signle-patient IND expanded access program (EAP) is designated 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 with vorasidenib.

Safety assessments (including vital signs, hematology, and serum chemistry) occur every two weeks for the first two cycles (28 day each cycle), then monthly for the duration of treatment. Treatment with vorasidenib will continue until, in the clinical judgement of the treating physician, the patient is no longer benefiting from treatment, vorasidenib is approved and available by prescription, or the study is terminated.

Request by treating physicians to file a single patient investigational new drug application as part of the expanded access program for vorasidenib will be considered on a case-by-case basis.

Requesting Expanded Access

All requests for Expanded Access must be made by an appropriately licensed treating physician on behalf of the patient. Servier, will in its sole discretion, evaluate requests for EA. Servier may consider, without limitation, some of all of the following criteria:

Inclusion Criteria

1. Age 12 or older and weighing at least 40 Kg.
2. Have Grade 2 or 3 oligodendroglioma or astrocytoma per WHO 2016 or 2021 criteria. Patients with Grade 4 astrocytomas may be considered on a case-by-case basis.
3. Having confirmed IDH1 or IDH2 gene mutation confirmed by tissue-based diagnosis.
4. Have at least 1 prior surgery for glioma (biopsy, sub-total resection, gross-total resection).
5. Have received chemotherapy and/or radiotherapy.
6. Have disease recurrence or progression. Patients with stable residual disease after standard of care therapy who, in the opinion of the treating physician, are likely to gain benefit from treatment may be considered on a case-by-case basis.
7. Have adequate bone marrow function.
8. Have adequate renal function.
9. Have adequate cardiac function.

Exclusion Criteria

1. Patient is eligible for ivosidenib Patient Assistance Program.
2. Patient is eligible for a clinical trial with vorasidenib or ivosidenib.
3. Prior treatment with IDH inhibitor, unless there is isoform switching confirmed by tissue-based diagnosis.
4. Have a heart-rate corrected QT interval using Fridericia's formula (QTcF) >/ 450msec or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome). Subjects with bundle branch block and prolonged QTcF may be eligible at the discretion of Servier Pharmaceuticals and the treating physician.
5. Are pregnant or breastfeeding.

A treating physician may submit questions or requests for EA by emailing vorasidenib.eap@earlyaccesscare.com

Patients, Caregivers and Patient Advocates: Requests for Expanded Access must be made by a qualified physician. Servier encourages patients and caregivers to have a candid conversation about reasons for considering an investigational medicine. Your physician will be required to supervise the treatment and comply with safety reporting responsibilities. Your physician must make the written request on your behalf.

Physicians and Healthcare Professionals: Only appropriately licensed physicians may make a written request on behalf of a patient. The physician must be qualified to administer and oversee the investigational treatment and comply with Servier safety reporting responsibilities. Investigational Review Board (IRB) approval for the use of the investigational medication is required. The physician must be willing to meet and follow all applicable legal and regulatory requirements. The physician will be required to provide adequate medical history and treatment information for a specific patient, obtain informed consent, and may be required to provide additional data on the progress and safety throughout the expanded access treatment interval.

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Available in These Countries:

• United States of America

SNDX-5613

SNDX-5613 is a highly selective inhibitor of the Menin-MLL binding interaction. Pivotal trials of SNDX-5613 are ongoing.

Clinical Trials At this time, Syndax Pharmaceuticals is enrolling patients to the AUGMENT-101 study, a phase 1-2 trial in patients with MLLr or mNPM1 acute leukemias. If you are interested in learning more about enrolling your patient to AUGMENT-101, contact SYNDAX at clinicaltrial@syndax.com.

For patients not eligible for the clinical trial, or if a clinical trial is not available, Expanded Access (sometimes called compassionate use) may be a path for patients to receive SNDX-5613.

Requesting Expanded Access All requests for Expanded Access must be made by a treating physician on behalf of the patient.. Syndax Pharmaceuticals has opened an Expanded Access Program for SNDX-5613.. A treating physician may submit questions or requests regarding Expanded Access by emailing SyndaxEAP@earlyaccesscare.com or by calling Early Access Care at 203-441-7939 Ext 185 and speaking with an Early Access Coordinator. Patients must provide informed consent for Expanded Access. No patient is guaranteed entry into the program solely by applying. Requests will be acknowledged within one business day.

Physicians must follow local laws and regulations appropriate for the country originating the request.

In the U.S. physicians may find additional information on Expanded Access by visiting the U.S. Food and Drug Administration website Expanded Access: Information for Physicians.

Physicians in other countries must follow regulations specific to the country health authorities.

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Available in These Countries:

• United States of America
• Canada
• France
• Israel
• Lithuania
• United Kingdom

MT1621

Expanded Access

Modis Therapeutics is committed to developing MT1621 for patients with thymidine kinase 2 deficiency (TK2d). Well designed clinical trials are the best way to determine the safety and efficacy of an investigational therapy. Clinical trial recruitment is essential in completing these clinical trials for the approval of a drug from regulatory authorities, such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). When a Modis investigational medicine is in clinical development, we encourage patients to speak with their treating physician regarding their eligibility to enroll in our clinical trials.

We acknowledge there are patients with TK2d who do not qualify for enrollment in a currently active clinical trial, but their treating physician feels he/she may benefit from access to MT1621. Therefore, Modis will evaluate, on a case-by-case basis, all requests for access to MT1621. The requesting physician will be required to provide adequate medical history, including treatments, for the specific patient, by completing the intake form. Making a request does not guarantee the granting of access to MT1621. We aim to respond to all requests with appropriate urgency. In general, a written response will be provided to the requesting physician within 14 days of receipt of all medical documentation required to assess patient eligibility.

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Available in These Countries:

• United States of America
• Argentina
• Austria
• Brazil
• Chile
• China
• Costa Rica
• France
• Germany
• Ghana
• India
• Italy
• Lebanon
• Mexico
• Montenegro
• Romania
• Spain
• Turkey
• United Kingdom

Zanidatamab

Zanidatamab is an investigational drug. It is a novel humanized, bispecific antibody that binds to the same 2 extracellular domains of HER2 as trastuzumab (ECD4) and pertuzumab (ECD2). Binding of zanidatamab results in blockade of ligand-dependent and independent growth and potent activation of antibody-dependent cellular cytotoxicity (ADCC). The unique binding geometry of zanidatamab also leads to increased tumor cell binding, receptor clustering, and receptor internalization relative to trastuzumab, including in settings of lower HER2 expression.

Expanded Access to Zanidatamab

ZWI-ZW25-EAP is an expanded access / compassionate use protocol, reviewed by the US Food and Drug Administration (FDA) and Health Canada. In addition, the protocol has been approved by an IRB. The objective of this protocol is to make zanidatamab available for patients with HER2-positive cancers who do not qualify for participation in, or who are otherwise unable to access an ongoing clinical study for zanidatamab. Find out more information about the zanidatamab EAP on ClinicalTrials.gov (NCT04578444)

Enrollment to ZWI-ZW25-EAP is available for patients not eligible for zanidatamab clinical trials. Currently, the EAP is available in Canada and the United States.

Safety Reporting

A written description of any serious adverse event, using the Zymeworks SAE report form, must be sent by email within 24 hours after awareeness of the event:

• Zanidatamab Pregnancy Report Form
• Zanidatamab Safety Report Form

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Available in These Countries:

• United States of America
• Canada
• France
• Italy
• Spain
• United Kingdom