The Latest News About Palforzia©

Amidst all the celebrations that an effective new treatment for peanut allergies was approved by the Food and Drug Administration, high cost is a major concern. Called Palforzia©, the medication is very simply an exactly measured dose of peanut flour (!) in a very expensive capsule. Previously the only treatment was avoidance and then hoping that epinephrine was on hand after accidental ingestion. Read the article below and plan to have a conversation with your allergist to see if this new therapy might be right for your child. Hopefully insurance companies will provide adequate coverage for this important treatment.

AR101 Palforzia capsules

The First Treatment Approved by the FDA for Peanut Allergies

Edited by Jon Terry
February 10, 2020

The Food and Drug Administration has approved Palforzia©, the first medication for treatment of peanut allergies. Palforzia is the brand name of an oral immunotherapy (OIT) formerly known as AR101 while it was being developed and undergoing clinical trials.

Aimmune Therapeutics, the company that has developed and tested Palforzia, states that the medication is intended to treat children ages 4 to 17 that have been diagnosed with a peanut allergy. It’s estimated that more than 1 million children (2.2%) in the United States have peanut allergies. Until now, parents who wanted keep their kids safe have had very few options.

“For peanut allergies, the treatment has always been avoidance. Just avoid, avoid, avoid — and then use epinephrine for when you have anaphylaxis,” explained Dr. Jonathan Spergel, chief of the Allergy Program at Children’s Hospital of Philadelphia, one of the research sites for the new medication. “This therapy is a big deal,” Spergel said, “because it’s the first treatment beyond avoidance.”

“Most allergists trained at a time when we never thought we’d see a treatment for food allergy,” said AAAAI President David M. Lang, MD, FAAAAI, who is also Chair of the Allergy/Immunology Department in the Respiratory Institute at Cleveland Clinic.

“Previously we recommended carrying injectable epinephrine and strict avoidance, which in some cases leads to serious or even life-threatening allergic reactions with inadvertent food exposures. Now, we have the first FDA approved treatment for food allergy, that when used in combination with avoidance measures, can reduce the possibility that an inadvertent exposure will lead to a serious allergic reaction.”

“The approval is monumental,” said Dr. Christina Ciaccio, chief of allergy and immunology at the University of Chicago. Before having an FDA-sanctioned product, Ciaccio said all she could tell young patients with peanut allergy was to avoid all exposures to the legume, but there was no way to treat the condition. “It’s just a landmark moment that we can finally have something to offer” as a desensitizing therapy.

OIT is not a cure for peanut allergy, and it is likely an individual starting this therapy will need to remain on it indefinitely. Patients utilizing this therapy will still be expected to avoid dietary peanuts and carry an epinephrine auto-injector.

Palforzia is designed to reduce the frequency and severity of allergic reactions for those suffering with peanut allergy who may accidentally be exposed to peanuts. The aim is, essentially, to make children slightly less allergic by reducing the reaction they have when exposed to peanuts.

Medical research studies have shown that a majority of those who reached maintenance on Palforzia could eat two peanut’s worth of peanut protein without reacting. This significantly reduces the risk of severe reaction if an allergic child accidentally eats some peanut-containing food. Aimmune is now studying similar protocols for egg allergy and tree nut allergies. 

“The goal is to raise your threshold,” said Dr. Scott Sicherer, director of the Elliot and Roslyn Jaffe Food Allergy Institute at the Icahn School of Medicine at Mount Sinai — also one of the sites where the drug was tested. So, if your child currently has an allergic reaction when he or she is exposed to the equivalent of 1/10th of a peanut, Shicherer explained, the goal of this medication would be to get to a point where he or she can handle one whole peanut without an allergic reaction.

The treatment consists of a strategy that has been used in food allergy circles for years: oral immunotherapy. By slowly and carefully exposing the body to increasing amounts of an allergen — in this case, peanuts — the treatment raises the threshold at which a reaction occurs. It’s not a one-time thing.

Palforzia is provided in capsule form that contains one active ingredient—a precise dose of peanut flour. The patient pulls the capsule apart and spreads the contents on food. 

Aimmune describes their drug as a standardized, pharmaceutical-grade product that employs Good Manufacturing Processes (GMP). GMP ensures the product is consistently produced and controlled according to industry-recognized quality standards. This means that all allergists that choose to administer the therapy will be using the identical formulation.

Allergists that currently offer peanut OIT either produce the peanut flour in their own labs or purchase it from third parties that formulate the doses for them.  

Parents should plan on bringing their child to the allergist for almost a full day, where they’ll start with the “teeniest” dose, Sicherer said — “like the dust of a peanut.” Kids will then be given incrementally bigger doses, about every 30 minutes, under close medical supervision. The next day, kids come back to take another dose in front of the doctor and be watched some more. Then kids come in every two weeks to get slightly higher doses.

In between those every-other-week visits — which Sicherer estimated will probably take at least two hours — the patient takes the medication daily at home. It comes in powder form in a capsule that is added to semi-solid foods like pudding or applesauce. A child goes through 11 increasing dose levels, and then may begin the daily maintenance dose. Along the way, doctors will closely calibrate dosage and work with families if children have reactions. After the dose escalation period, which lasts six months or longer, the patient continues to take a daily therapeutic dose to maintain desensitization.

Again, it’s a process, not a one-and-done kind of deal.

Studies leading up to its approval showed some pretty serious potential side effects, like nausea, vomiting, hives, wheezing, and even anaphylaxis. It is essential that patients carry epinephrine at all times to treat any anaphylaxis emergencies.

Aimmune claims that of those that experienced reactions similar to anaphylaxis, 98.2% were classified as “mild” or “moderate” and included nausea, itchiness in the throat, and vomiting. Their research showed an almost tripling of the risk of an anaphylactic reaction (9.4%) during the time the patient is building tolerance to reach the maintenance dose compared with placebo (3.8%). 

You can’t buy Palforzia quite yet. Just because the FDA has approved the medication does not mean it’s available to patients. “The next step is when can your doctor go to the pharmacy and order it?” Spergel explained. “That typically takes a couple of months.” Aimmune expects allergists will be able to begin prescribing the medication within a matter of weeks.

There’s also the cost. The price for Palforzia has been set at $890 a month, or roughly $11,000 per year. The annual list price is higher than the $4,800 to $7,200 range recommended by ICER, a Boston-based independent panel that reviews drug pricing.

The list price of a drug is not necessarily what patients actually pay. Out-of-pocket costs vary based on the duration of the treatment and individual healthcare plans. Again, because the medication is new, insurance coverage is still very much in the works.

The other issue for families is how quickly a medication like this will be approved by insurers, which can also take time.

Palforzia may soon face competition from French drug maker DBV Technologies SA, which has filed for U.S. approval of a once-daily skin patch to reduce sensitivity to peanut exposure.

DBV Technologies resubmitted a BLA for their Viaskin Peanut therapy this August. Viaskin Peanut is an immunotherapy “patch” that desensitizes the immune system by introducing small amounts of peanut allergen via the skin rather than orally.

The company has just begun enrolling patients for a Phase 2 clinical trial of AR201 — a therapeutic candidate for egg allergy —and is working on an as yet unnamed therapy for allergy to tree nuts. The FDA is expected to make its approval decision for DBV's Viaskin Peanut patch by August 2020. 

OIT will not be right for every patient. It is not a “one size fits all” approach. How to predict which individuals will respond to OIT and which individuals will be at highest risk of side effects (including anaphylaxis) has not been clearly determined, and there are many other important questions about OIT that require ongoing study.

But Sicherer said Palforzia might still be an appealing option for parents of children who have experienced accidental exposures to peanuts, despite their best efforts to avoid, avoid, avoid. “You know, they’re trying to be careful, they’re talking to restaurants, but they’ve ended up having allergic reactions anyway because their children are very sensitive to a small amount,” Sicherer said. The new drug “could be a great match,” he added, “for someone who wants to raise that threshold.”

All of that means parents should plan on having thorough discussions with their children’s health care providers weighing the risks and potential benefits, which makes this a highly personal decision, the experts emphasized.

The AAAAI encourages patients and families to engage with an allergist/immunologist to discuss the risks, benefits, and alternatives of OIT and all other emerging treatments for food allergy. Ultimately, the choice of treatment, including that of active non-intervention, will be based on individual and family factors after careful discussion with one’s physician.

“Patients and their families will need to understand the realities of these new therapies. As physicians, we must invest the time to explain the potential for benefit compared with the potential for harm/burden, and permit patients and families to express their values and preferences, allowing them to participate in the medical decision making process to determine whether they desire to proceed with food oral immunotherapy,” said Dr. Lang.

AAAAI’s Find an Allergist/Immunologist service will allow you to find a trusted AAAAI member specialist close to home.

You can learn more about the current state of OIT at the AAAAI’s website, aaaai.org.

Reporting by Dave Bloom for SnackSafely.com; Carl O'Donnell for Reuters.com; Catherine Pearson for HUFFPOST.com

Sources:

AR101 for Peanut Allergy — Aimmune Therapeutics

Development Pipeline — Aimmune Therapeutics

Aimmune Therapeutics’ Pivotal Phase 3 PALISADE Trial of AR101 Meets Primary Endpoint in Patients With Peanut Allergy — Aimmune Press Release

FDA Committee Recommends Approval For New Peanut Allergy Treatment — National Public Radio

For Children With Peanut Allergies, F.D.A. Experts Recommend a New Treatment — New York Times

https://snacksafely.com/2019/09/faq-for-palforzia/

https://www.huffpost.com/entry/peanut-allergy-drug-palforzia-fda_l_5e39902fc5b687dacc739467

http://news.trust.org/item/20200131230744-u4k3o/

https://snacksafely.com/2020/02/what-you-need-to-know-about-the-first-peanut-allergy-treatment-approved-by-the-fda/?utm_medium=onesignal

© Copyright Allergy Advocacy Association 2020 

The information provided on this site is in no way intended to be a substitute for medical advice,
diagnosis, or treatment with a licensed physician.
The Allergy Advocacy Association is a 501(c)(3) non-profit, tax-exempt organization.
Copyright 2020 © Allergy Advocacy Association, Inc. All rights reserved.  Terms & Conditions