Aimmune announced the U.S. FDA has approved Palforzia [Peanut (Arachis hypogaea) Allergen Powder-dnfp], making this the first approved treatment for patients with a confirmed diagnosis of peanut allergy. Palforzia is an oral immunotherapy indicated for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut. Palforzia is to be used in conjunction with a peanut-avoidant diet. It is not indicated for the emergency treatment of allergic reactions, including anaphylaxis.
The FDA approval of Palforzia was based on seven clinical studies, including the pivotal Phase 3 PALISADE and RAMSES clinical trials.
PALISADE (Peanut ALlergy oral Immunotherapy Study of AR101 for DEsensitization in children and adults) was an international, randomized 3:1, double-blind, placebo-controlled trial of the efficacy and safety of AR101 in a Characterized Oral Desensitization ImmunoTherapy (CODIT) approach in patients with peanut allergy. PALISADE enrolled 554 peanut-allergic patients ages 4–49, all of whom had to experience dose-limiting symptoms at or before the 100-mg dose of peanut protein in an entry double-blind, placebo-controlled food challenge (DBPCFC), which allowed consecutive doses of 1, 3, 10, 30 and 100 mg of peanut protein, given 20 to 30 minutes apart. Patients enrolled in PALISADE underwent a dose escalation period of approximately 22 weeks to reach a maintenance dose of 300 mg per day of AR101 or placebo, then continued with daily maintenance at 300 mg per day of AR101 or placebo for approximately six months. At the end of the maintenance period, patients underwent an exit DBPCFC, which tested consecutive doses of 3, 10, 30, 100, 300, 600 and 1000 mg of peanut protein, given 20 to 30 minutes apart, as tolerated with no or only mild symptoms. The study met the primary efficacy endpoint, as 67.2% of AR101 patients ages 4–17 tolerated at least a 600-mg dose of peanut protein in the exit food challenge, compared to 4.0% of placebo patients. 50.3% of AR101 patients ages 4–17 tolerated a 1000-mg dose of peanut protein in the exit food challenge, compared to 2.4% of placebo patients. Among patients ages 4–17 who completed treatment with AR101, 96.3% tolerated a 300-mg dose of peanut protein in the exit food challenge, 84.5% tolerated a 600-mg dose, and 63.2% tolerated a 1000-mg dose. 79.6% of AR101 patients ages 4–17 completed the trial; of the 20.4% who discontinued treatment, 12.4% withdrew due to treatment-related adverse events.
AR101 is a novel, investigational oral biologic drug for use in oral immunotherapy (OIT) in patients with peanut allergy. The drug, which is manufactured in accordance with current Good Manufacturing Practices (cGMP), has a characterized protein profile found in peanuts, analyzed to ensure consistent major allergen content. The amount of active ingredient in each AR101 capsule is controlled to ensure minimal variability of allergen content across doses of a given strength.