Company introduces Risk Evaluation and Mitigation Strategy program for Patients, Healthcare Providers, Pharmacies, and Healthcare Facilities
As we reported, Aimmune Therapeutics received FDA approval of their peanut allergy oral immunotherapy (OIT) drug on January 31, the first-ever drug to receive FDA approval for the treatment of food allergy.
OIT is a process where minute quantities of an allergen are introduced orally and gradually increased as the therapy progresses. Treatment with Palforzia involves separating a capsule or opening a sachet of the drug over food with the dosage of active ingredient increased over time. The exposure to increasing doses of the allergen over time desensitizes the patient, with the goal being the patient will be able to tolerate small, inadvertent exposure to peanuts such as might occur through accidental cross-contact.
OIT in general and Palforzia in particular are not considered cures for the food allergies they treat, though they can provide significant protection from anaphylaxis when a patient that has undergone the up-dosing course of the therapy is inadvertently exposed to those allergens.
Because OIT is predicated upon exposing the patient to small quantities of the proteins they are allergic to, there are risks of reactions associated with treatment including anaphylaxis. The FDA seeks to mitigate that risk for Palforzia by mandating a program that must be followed by patients, prescribers, pharmacies, and healthcare settings.
Aimmune describes the program this way:
The PALFORZIA REMS (Risk Evaluation and Mitigation Strategy) Program is a safety program that manages the risk of anaphylaxis associated with PALFORZIATM. The PALFORZIA REMS Program is required by the Food and Drug Administration (FDA) to ensure the potential benefits of PALFORZIA outweigh its risks.
The goal of the PALFORZIA REMS Program is to mitigate the risk of anaphylaxis associated with PALFORZIA by:
- Ensuring that healthcare providers who prescribe and healthcare settings that dispense and administer PALFORZIA are educated on the following:
- the risk of anaphylaxis associated with the use of PALFORZIA
- the Initial Dose Escalation and first dose of each Up-Dosing level must only be administered to patients in a healthcare setting equipped to monitor patients, and to identify and manage anaphylaxis.
- Ensuring that the Initial Dose Escalation and the first dose of each Up-Dosing level of PALFORZIA are only dispensed and distributed to certified healthcare settings and only administered to patients in certified healthcare settings.
- Ensuring that PALFORZIA is only dispensed and administered to patients who are informed, by enrolling in the PALFORZIA REMS Program, of the need to have injectable epinephrine available for immediate use at all times, the need for monitoring with the Initial Dose Escalation and first dose of each Up-Dosing level, the need for continued dietary peanut avoidance, and how to recognize the signs and symptoms of anaphylaxis.
To undergo treatment with Palforzia, the patient — presumably via their parent or guardian as the therapy is currently only approved for children 4 through 17 years — must first enroll in the REMS program. Here are the instructions for doing so:
- DISCUSS THE BENEFITS AND RISKS of PALFORZIA with your doctor.
- ASK YOUR DOCTOR any questions you have about taking PALFORZIA and about the PALFORZIA REMS Program.
- MAKE SURE you understand:
- How to enroll and take part in the PALFORZIA REMS
- The benefits and risks of PALFORZIA
- That you must have injectable epinephrine available at all times
- That you must avoid peanuts or peanut containing foods in the diet
- That you know the signs and symptoms of severe allergic reaction (anaphylaxis) and to tell your doctor if you have any of these signs or symptoms
- You will need to receive certain doses at your doctor’s office
- You will need to be monitored after doses received at your doctor’s office for at least 60 minutes
- TOGETHER WITH YOUR DOCTOR, complete and sign the Patient Enrollment Form.
Assuming you have enrolled in the REMS program and are prescribed the medication, the enclosed Medication Guide for Palforzia provides important “need to know” information regarding the medication in a Q&A fashion. Here is the information provided in answer to the question “What is the most important information I should know about PALFORZIA?“:
PALFORZIA can cause severe allergic reactions called anaphylaxis that may be life-threatening.
- You will receive your first dose in a healthcare setting under the observation of trained healthcare staff.
- You will receive the first dose of all dose increases in a healthcare setting.
- In the healthcare setting, you will be observed for at least 1 hour for signs and symptoms of a severe allergic reaction.
- If you have a severe reaction during treatment, you will need to receive an injection of epinephrine immediately and get emergency medical help right away.
- You will return to the healthcare setting for any trouble tolerating your home doses.
Stop taking PALFORZIA and get emergency medical treatment right away if you have any of the following symptoms after taking PALFORZIA:
- Trouble breathing or wheezing
- Chest discomfort or tightness
- Throat tightness
- Trouble swallowing or speaking
- Swelling of your face, lips, eyes, or tongue
- Dizziness or fainting
- Severe stomach cramps or pain, vomiting, or diarrhea
- Hives (itchy, raised bumps on skin)
- Severe flushing of the skin
For home administration of PALFORZIA, your doctor will prescribe injectable epinephrine, a medicine you must inject if you have a severe allergic reaction after taking PALFORZIA. Your doctor will train and instruct you on the proper use of injectable epinephrine.
Talk to your doctor and read the epinephrine patient information if you have any questions about the use of injectable epinephrine.
PALFORZIA is only available through a restricted program called the PALFORZIA Risk Evaluation and Mitigation Strategy (REMS) Program. Before you can receive PALFORZIA, you must:
- Enroll in this program.
- Receive education about the risk of a severe allergic reaction (anaphylaxis) by a healthcare provider who practices in a setting that is certified through the REMS program.
- Understand that you will be monitored in a healthcare setting during and after the Initial Dose Escalation and for the first dose of each Up-Dosing level (see Dosing Information).
- Receive education about how to maintain a peanut-free diet. You must attest that you will continue to avoid peanuts at all times.
- Fill the prescription your healthcare provider gives you for the injectable epinephrine. You must attest that epinephrine will be available to you at all times.
Talk to your healthcare provider for more information about the PALFORZIA REMS program and how to enroll.
We encourage you to read the entire Medication Guide Q&A which provides additional need-to-know information.
If you are considering Palforzia for treatment of peanut allergy, the first step is to discuss the program with your allergist who may or may not already be enrolled in the Palforzia REMS program. If not and your allergist does not plan on enrolling, you may be instructed to find an allergist who is enrolled.
Here are important links where you can find additional information:
- Welcome to the PALFORZIA REMS (Risk Evaluation and Mitigation Strategy) Program — Aimmune Therapeutics
- Palforzia Home Page — Aimmune Therapeutics
- Palforzia for Healthcare Providers Home Page — Aimmune Therapeutics
- Palforzia Medication Guide — Aimmune Therapeutics