FDA Approves Palforzia: The First Drug Developed for the Treatment of Peanut Allergy in Children

Last week, the U.S. Food and Drug Administration (FDA) approved Palforzia [Peanut (Arachis hypogaea) Allergen Powder-dnfp], a powder that reduces allergic reactions, including anaphylaxis, that may occur following accidental exposure to peanuts. Palforzia can be administered to children ages 4 through 17 with a confirmed diagnosis of peanut allergy. However, children taking Palforzia must continue to avoid consuming peanuts.

The efficacy of Palforzia was assessed in a randomized, double-blind, placebo-controlled study conducted in the U.S., Canada and Europe comprised of approximately 500 peanut-allergic individuals. Researchers evaluated the percentage of study participants tolerating an oral dose of 600 mg of peanut protein (twice the daily maintenance dose of Palforzia) with no more than mild allergic symptoms after 6 months of maintenance treatment. According to the study results, 67.2% of Palforzia recipients tolerated a 600 mg dose of peanut protein, juxtaposed to 4% of recipients in the placebo arm.

Palforzia’s safety was tested in two double-blind, placebo-controlled studies comprised of approximately 700 peanut-allergic individuals. The results showed that the most common side effects of Palforzia were:

  • Abdominal pain
  • Vomiting
  • Nausea
  • Tingling in the mouth
  • Itching
  • Cough
  • Runny nose
  • Throat irritation and tightness
  • Hives
  • Wheezing and shortness of breath
  • Anaphylaxis

The FDA noted that Palforzia should not be administered to patients with uncontrolled asthma.

Three Phase Treatment

Treatment with Palforzia, which is manufactured from peanuts and packaged in pull-apart color-coded capsules, consists of three phases: Initial Dose Escalation, Up-Dosing, and Maintenance. The Initial Dose Escalation phase is administered on a single day. The Up-Dosing phase includes 11 increasing dose levels and occurs over several months. The FDA noted that both the initial Dose Escalation, and the first dose of each Up-Dosing level, are administered under supervision of a healthcare professional in a healthcare setting with the ability to manage potentially severe allergic reactions, including anaphylaxis. Also, while anaphylaxis may occur at any time during Palforzia therapy, patients are at the highest risk during and after the Initial Dose Escalation and the first dose of each Up-Dosing level.

During the Up-Dosing phase, if a patient tolerates the first dose of an increased dose level, the patient may continue that dose level daily at home. Subsequent to the completion of all Up-Dosing levels, the patient may begin the daily maintenance dose. Any patient who experiences certain allergic reactions due to Palforzia may need to either discontinue treatment or have their dosing schedule modified.

To allay the risk of anaphylaxis associated with Palforzia, the FDA is requiring a Risk Evaluation and Mitigation Strategy (REMS) with this approval, which includes elements to assure safe use. Palforzia will only be available through specially certified healthcare providers, health care settings, and pharmacies to patients who are enrolled in the REMS program.

“Peanut allergy affects approximately 1 million children in the U.S. and only 1 out of 5 of these children will outgrow their allergy. Because there is no cure, allergic individuals must strictly avoid exposure to prevent severe and potentially life-threatening reactions,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research in an FDA press release.

“Even with strict avoidance, inadvertent exposures can and do occur. When used in conjunction with peanut avoidance, Palforzia provides an FDA-approved treatment option to help reduce the risk of these allergic reactions in children with peanut allergy.”