An Aimmune Therapeutics drug developed to treat peanut allergy won FDA approval late Friday—the first time the agency has approved an oral immunotherapy for a food allergy.
The Aimmune (NASDAQ: AIMT) drug, Palforzia, was developed to desensitize patients to peanuts. The FDA decision covers patients between the ages of 4 and 17 who have a confirmed peanut allergy diagnosis. Brisbane, CA-based Aimmune says it expects the drug will become available to patients in coming weeks.
Palforzia is a powder made from peanut flour that comes in capsules or packets. Aimmune developed its therapy so that each dose contains precise amounts of peanut flour and food proteins to ensure consistency. The therapy is meant to be taken daily with food. By increasing doses over time, patients become desensitized to peanuts.
The Aimmune therapy is not a peanut allergy cure. But cooking and manufacturing processes can introduce peanut proteins into foods even if peanuts are not listed as an ingredient, Chief Medical Officer Daniel Edelman said on a conference call. Desensitization from the Aimmune therapy can make allergic responses to those proteins less severe.
“While they still must practice avoidance, it can mitigate allergic reactions after accidental consumption,” Edelman said.
Palforzia was tested in a double-blind, placebo-controlled Phase 3 study that enrolled more than 500 patients. Peanut sensitivity was evaluated by having patients eat increasing amounts of peanut protein until the end of this “food challenge” or an allergic reaction that caused them to stop. The FDA said that 67.2 percent of patients who were treated with the Aimmune drug tolerated a 600 mg dose of peanut protein in the challenge. In the placebo group, 4 percent of patients could complete the challenge.
In September, an independent advisory panel voted to recommended that the FDA approve the Aimmune drug. In a separate vote, the body recommended that an approval come with additional safeguards to protect against health risks that patients might experience from treatment. FDA staff documents noted that allergic reactions and other side effects were observed in 90 percent of patients who participated in clinical trials. Twelve of those patients developed eosinophilic esophagitis, an accumulation of a type of white blood cell in the esophagus. This condition can cause chest pain and difficulty swallowing.
The FDA decision acknowledges the drug’s safety risks. The approval requires a “Risk Evaluation and Mitigation Strategy,” a safety plan to inform both patients and physicians about the risks. This program requires that patients starting treatment and those moving up to a higher dose do so in a healthcare setting where epinephrine, which can reverse symptoms of an allergic reaction, is available. The drug’s label also comes with a boxed warning to alert physicians and patients that the drug can cause anaphylaxis, a severe and potentially life-threatening allergic reaction. Such warnings are common for immunotherapies that treat allergic conditions.
Aimmune set an $890 a month wholesale price for Palforiza, before any rebates or discounts. That price is consistent for the updosing and maintenance phases. Because peanut allergy is a chronic condition, patients must continue taking the therapy in order to maintain peanut desensitization. But Chief Commercial Officer Andrew Oxtoby said that Aimmune will offer assistance programs to help patients cover the cost of the drug. The company will also make Palforiza available for free to those who are not covered by commercial insurance or Medicaid.
CEO Jayson Dallas added that an ongoing study is testing the therapy in children between 1 and 4. Aimmune has also submitted Palforzia for review by European regulators. The company has more food allergy immunotherapies in its pipeline, including AR-201, an experimental egg allergy therapy that is in Phase 2 testing. Dallas said that the company is also developing treatments for multiple tree nut allergies.
Photo by Aimmune Therapeutics