Approved Risk Evaluation and Mitigation Strategies (REMS)

Palynziq (pegvaliase-pqpz)

BLA #761079

REMS last update: 12/09/2023

• View the Palynziq Prescribing Information and Medication Guide at DailyMed.
• View Palynziq's Regulatory Information at Drugs@FDA

• Goals
• Summary
• Assessment Plan
• Update history

What is the purpose of the REMS?

The goal of the PALYNZIQ (pegvaliase-pqpz) REMS is to mitigate the risk of anaphylaxis associated with PALYNZIQ by:

1. Ensuring that prescribers are educated on the risk of anaphylaxis associated with the use of PALYNZIQ
2. Ensuring that prescribers are educated and adhere to the following:
   • Counsel patients on how to recognize and respond to signs and symptoms of anaphylaxis
   • Enroll patients in the PALYNZIQ REMS
   • Prescribe auto-injectable epinephrine with PALYNZIQ
3. Ensuring that PALYNZIQ is only dispensed to patients with documentation of safe use conditions
   • Patient education and enrollment in the PALYNZIQ REMS
   • Having auto-injectable epinephrine available at all timesM/li>
4. Ensuring that patients are educated on the following:
   • How to recognize and respond to signs and symptoms of anaphylaxis
   • The need to carry auto-injectable epinephrine with them at all times

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).

View application holder(s) REMS Website

Healthcare Providers who prescribe PALYNZIQ must:

To become certified to prescribe	Review the drug’s Prescribing Information. Review the following: Prescriber Guide and REMS Program Overview. Successfully complete the Prescriber Knowledge Assessment and submit it to the REMS. Enroll in the REMS by completing the Prescriber Enrollment Form and submitting it to the REMS.
Before treatment initiation (first dose)	Assess the patient’s need for an adult observer and for premedication as described in the Prescriber Guide. Counsel the patient on the risk for anaphylaxis, including how to recognize and respond to signs and symptoms of anaphylaxis and having autoinjectable epinephrine available at all times using the Patient Guide, Safety Video, and Wallet Card. Provide the patient with the Patient Guide and Wallet Card, and direct the patient to PALYNZIQREMS.com to view the Safety Video. Enroll the patient in the REMS by completing and submitting the Patient Enrollment Form. Provide a completed copy of the form to the patient and retain a completed copy in the patient’s record. Provide the patient with a prescription for auto-injectable epinephrine to accompany the prescription for PALYNZIQ.
During treatment; before each prescription	Assess the patient for anaphylaxis episodes. Assess the patient’s supply of auto-injectable epinephrine. Provide a prescription for auto-injectable epinephrine, if the patient’s supply is inadequate.
At all times	Report anaphylaxis episodes to the REMS. Report treatment discontinuation or transfer of care to the REMS.

Patients who are prescribed PALYNZIQ:

Before treatment initiation

Review the Patient Guide, Safety Video, and Wallet Card. Enroll in the REMS by completing the Patient Enrollment Form with the prescriber. Enrollment information will be provided to the REMS. Receive counseling from the prescriber on how to recognize and respond to signs and symptoms of anaphylaxis, and having auto-injectable epinephrine with you at all times using the Patient Guide, Safety Video, and Wallet Card. Adhere to the safe use conditions including obtaining auto-injectable epinephrine.

At all times

Inform the prescriber if you have a severe allergic reaction (anaphylaxis). Inform the prescriber if you need more auto-injectable epinephrine. Have auto-injectable epinephrine and the Wallet Card with you. Inform all doctors about this treatment. Report a change in prescriber or contact information to the REMS. Report treatment discontinuation to the REMS.

Pharmacies that dispense PALYNZIQ must:

To become certified to dispense	Designate an authorized representative to carry out the certification process and oversee implementation and compliance with the REMS on behalf of the pharmacy. Have the authorized representative review the REMS Program Overview. Have the authorized representative enroll in the REMS by completing and submitting the Pharmacy Enrollment Form. Establish processes and procedures to verify and document the patient has auto-injectable epinephrine and report anaphylaxis episodes to the REMS/manufacturer/prescriber. Train all relevant staff involved in the dispensing of PALYNZIQ on the requirements using the REMS Program Overview.
Before Dispensing	Verify and document that the patient has auto-injectable epinephrine through the processes and procedures established as a requirement of the REMS. Obtain authorization to dispense each prescription by contacting the REMS to verify prescriber certification and patient enrollment.
To maintain certification to dispense	Have the new authorized representative enroll in the REMS by completing the Pharmacy Enrollment Form if the authorized representative changes.
At all times	Report anaphylaxis episodes to the REMS. Not distribute, transfer, loan, or sell PALYNZIQ, except to certified dispensers. Maintain records documenting staff’s completion of REMS training. Maintain records that all processes and procedures are in place and are being followed. Comply with audits carried out by BioMarin Pharmaceutical Inc. or a third party acting on behalf of BioMarin Pharmaceutical Inc. to ensure that all processes and procedures are in place and are being followed.

Wholesalers-distributors that distribute PALYNZIQ 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 distributing on the REMS requirements.
At all times	Distribute only to certified pharmacies and maintain records of all distributions. Comply with audits carried out by BioMarin Pharmaceutical Inc. or a third party acting on behalf of BioMarin Pharmaceutical Inc. to ensure that all processes and procedures are in place and are being followed.

Assessment Plan

This webpage provides REMS assessment plan measures, metrics, and indicators for drug and biological products with a REMS. The REMS assessment plan measures, metrics, and indicators are part of the overall REMS assessment plan used by FDA to evaluate the performance of a REMS and are included in FDA approval letters. The current REMS assessment plan measures, metrics, and indicators are extracted from regulatory letters that contain REMS information, including FDA approval letters, and posted here as they become available.

What updates have been made to the REMS?

Date	Summary of change
12/09/2023	Modified to change the timetable for submission of assessments of the REMS from yearly to every two years.
10/06/2020	Modified to: Align with prior approval supplements S-005,S-006, and S-007 Update logo Reflect formatting changes that attention to the safety video Clarify the pharmacy enrollment process Add Spanish language materials for prescribers, pharmacies, and patients
05/24/2018	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.