Approved Risk Evaluation and Mitigation Strategies (REMS)

Juxtapid (lomitapide)
NDA #203858
REMS last update: 05/27/2021



What is the purpose of the REMS?

The goal of the JUXTAPID REMS is to mitigate the risk of hepatotoxicity associated with the use of JUXTAPID by ensuring that:

  1. Prescribers are educated about the approved indication for JUXTAPID, the risk of hepatotoxicity associated with the use of JUXTAPID; and the need to monitor patients during treatment with JUXTAPID as per product labeling.
  2. JUXTAPID is dispensed only to patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia (HoFH).
  3. Patients are informed about the risk of hepatotoxicity associated with the use of JUXTAPID and the need for baseline and periodic monitoring.

What do participants need to know?

Below is a general overview of the REMS for all REMS participants (e.g., patients, pharmacies, and healthcare providers). See the application holder(s) REMS Website or the approved REMS materials for more information.

View application holder(s) REMS Website See Disclaimer page regarding links to external sites

Healthcare Providers who prescribe Juxtapid must

To become certified to prescribe
During treatment; before each prescription
Before treatment inititation
  • Counsel the patient on the appropriate use and risks associated with Juxtapid using the Patient Guide. Provide a copy of the material to the patient.
    | Patient Guide |
  • Enroll the patient by completing and submitting the Patient-Prescriber Acknowledgement Form to the REMS Program.
    | Patient-Prescriber Acknowledgement Form |

Pharmacies that dispense Juxtapid must

To become certified to dispense
  • Train all relevant staff involved in dispensing on the REMS program requirements using the Pharmacy Training Module.
    | Pharmacy Training Module and Knowledge Assessment |
  • Establish processes and procedures to ensure pharmacy staff obtain authorization to dispense each prescription by accessing the REMS Program database or calling the REMS Program Coordinating Center.
  • Designate an authorized representative to carry out the certification process on behalf of the pharmacy.
  • Have the authorized representative review the drug's Prescribing Information.
  • Have the authorized representative review the Program Fact Sheet.
    | Fact Sheet |
  • Have the authorized representative successfully complete the Knowledge Assessment that is included in the Pharmacy Training Material, and submit it to the REMS Program.
    | Pharmacy Training Module and Knowledge Assessment |
  • Have the authorized representative enroll in the REMS Program by completing the Pharmacy Enrollment Form.
    | Pharmacy Enrollment Form |
Before dispensing
  • Obtain authorization to dispense each prescription by accessing the REMS Program database or calling the REMS Program Coordinating Center to verify the completion of the Patient-Prescriber Acknowledgement Form, certification of the prescriber, and receipt of the Prescription Authorization Form for each new prescription.
To maintain certification to dispense, if the pharmacy designates a new authorized representative
  • Designate an authorized representative to carry out the certification process on behalf of the pharmacy.
  • Have the authorized representative review the drug's Prescribing Information.
  • Have the authorized representative review the Program Fact Sheet.
    | Fact Sheet |
  • Have the authorized representative successfully complete the Knowledge Assessment that is included in the Pharmacy Training Material, and submit it to the REMS Program.
    | Pharmacy Training Module and Knowledge Assessment |
  • Have the authorized representative enroll in the REMS Program by completing the Pharmacy Enrollment Form.
    | Pharmacy Enrollment Form |
At all times
  • Maintain records of training, prescription data, and documentation that all processes and procedures are in place and are being followed and provide upon request to Amryt, FDA, or a third party acting on behalf of Amryt or FDA.
  • Comply with audits carried out by Amryt, FDA, or a third party acting on behalf of Amryt, or FDA to ensure that all proccesses and procedures are in place and are being followed.

Patients who are prescribed Juxtapid must

Before the first prescription
  • Review the Patient Guide.
    | Patient Guide |
  • Enroll in the REMS Program by completing the Patient-Prescriber Acknowledgement Form with the prescriber. Enrollment information will be provided to the REMS Program.
    | Patient-Prescriber Acknowledgement Form |
  • Receive Counseling from the prescriber on the risk of liver toxicity, periodic liver function monitoring, and appropriate patient selection.

Wholesalers that distribute Juxtapid must

To be able to distribute
  • Establish processes and procedures to ensure that the drug is distributed only to certified pharmacies.
  • Train all relevant staff involved in distribution on the REMS Program Requirements.
At all times
  • Maintain records of distribution data of all shipments and documentation to support that all processes and procedures are in place, are being followed, and make the documentation available for audits.
  • Comply with audits carried out by Amryt, FDA, or a third party on behalf of Amryt or FDA to ensure that all processes and procedures are in place and are being followed.

What materials are included in the REMS?

The REMS includes a REMS Document. In addition, the REMS includes the following materials intended for patients and healthcare providers. For a specific Medication Guide of a product in the Juxtapid REMS, see the DailyMed link(s).
Material Name Material Name Link
Fact Sheet (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Fact_Sheet.pdf
Letter for Healthcare Providers (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Letter_For_Healthcare_Providers.pdf
Letter for Pharmacists (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Letter_For_Pharmacists.pdf
Patient Guide (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Patient_Guide.pdf
Patient-Prescriber Acknowledgement Form (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Patient-Prescriber_Acknowledgement_Form.pdf
Pharmacy Enrollment Form (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Pharmacy_Enrollment_Form.pdf
Pharmacy Training Module and Knowledge Assessment (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Pharmacy_Training_Module_and_Knowledge_Assessment.pdf
Prescriber Enrollment Form (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Prescriber_Enrollment_Form.pdf
Prescriber Training Module and Knowledge Assessment (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Prescriber_Training_Module_and_Knowledge_Assessment.pdf
Prescription Authorization Form (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_Prescription_Authorization_Form.pdf
REMS Document (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_REMS_Document.pdf
REMS Full (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_REMS_Full.pdf
REMS Website Screenshots (PDF) https://www.accessdata.fda.gov/drugsatfda_docs/rems/Juxtapid_2021_05_27_REMS_Website_Screenshots.pdf

What updates have been made to the REMS?

Date Summary of change
05/27/2021 Modified to add online fillable fields in REMS forms and modified the design and formatting of the REMS materials.
10/21/2020 Revised to make editorial changes.
05/18/2020 Revised to reflect change in ownership.
03/13/2020 Revised to make editorial changes.
06/13/2019 Revised to update the corporate address.
06/18/2018 Revised to make an editorial change.
06/04/2018 Modified to:

  1. remove the option of emailing completed Patient-Prescriber Authorization Forms (PPAFs) for protection of patient information
  2. require patients reaching age 18 to sign a PPAF (previously signed by a parent)
  3. add language on the website to clarify the order of actions for prescriber participation in the REMS
  4. establish an all-electronic process for submission of the certificate of completion of training and knowledge assessment
  5. streamline the process for completion of the prescriber enrollment form (use of auto-completion of the form using prescriber information already submitted)
  6. add text to the REMS website requesting pharmacies to contact Aegerion before attempting to certify
  7. update font colors on REMS forms to enhance readability
  8. add clarifying language on the Prescription Authorization Form (PAF) regarding how to submit a prescription
03/02/2017 Revised to make an editorial change.
01/03/2017 Modified to conform to the Safety Labeling Change Notification/REMS Modification Notification letter issued on March 11, 2016 in the REMS document, the existing REMS materials, and the following new REMS materials: Patient Guide and Patient-Prescriber Acknowledgement Form.
08/13/2013 Modified to allow prescribers to complete the REMS-required training and enrollment online.
12/21/2012 Approval of the REMS.

Disclaimer: This webpage provides general information about REMS programs to various REMS participants (e.g., patients, pharmacies, and healthcare providers). The summary information provided herein is not comprehensive and may not include all of the information relevant to REMS participants. This webpage does not constitute a replacement, modification, or revision of the approved REMS document, including any appended REMS materials. Refer to the approved REMS document for complete information on the REMS requirements for each approved application.

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