The U.S. FDA has approved Palforzia, the oral immunotherapy (OIT) for peanut allergy, for treatment in toddlers with peanut allergy.
When it was initially approved for patients ages 4 to 17 years in 2020, the treatment became the first-ever FDA-approved food allergy therapy. The intent of Paliforzia is to gradually desensitize a patient, to protect against the risk of allergic reactions due to accidental exposure to peanut.
The FDA’s July 2024 approval now expands Palforzia’s prescribing indication to peanut-allergic children ages 1 to 3 years old.
“I’m overjoyed that there’s now an option for a very important age group,” says Dr. Stephen Tilles, an allergist in Washington state. Tilles is the former senior medical director at Nestlé Health Science’s Aimmune Therapeutics and a co-author on the study that led to the approval.
Stallergenes Greer, a global healthcare company, acquired Palforzia in September 2023 from Nestlé. Dr. Elena Rizova, medical director of the firm, expressed delight at the approval.
“There is a high unmet medical need for this age group,” she said in a press release. “We are confident that this indication extension will alleviate the burden of peanut allergy for younger patients and their families.”
Tilles says there are advantages with starting the therapy at a young age. “Typically before they turn 4, the young immune system is much more amenable to being fundamentally changed,” he explains.
Patients on the treatment take daily doses of Palforzia, in gradually increasing amounts, until a maintenance level is reached. The doses come in capsules that contain calibrated amounts of peanut protein powder that’s mixed into soft food.
Palforzia OIT for toddlers is expected to launch in early 2025, according to Stallergenes Greer. At that time, enrollment for the Palforzia co-pay program will become available for those patients.
Research on Palforzia for Toddlers
The treatment’s regulatory approval for toddlers is based on findings from the Phase 3 Poseidon study. Results of the study, which included 146 peanut-allergic toddlers, were published in October 2023 in the New England Journal of Medicine Evidence.
The findings for toddlers showed “impressive efficacy and safety,” says Tilles, who serves as a consultant on Palforzia.
Patients ages 1 to 3 took either Palforzia (98 patients) or placebo doses (48 patients) daily for 12 months in the double-blind clinical trial. Participants on the treatment received gradually increasing daily doses up to 300 milligrams of Palforzia powder (equivalent to one peanut). They then continued on that as the maintenance dose.
Tilles is encouraged by the level of tolerance participants showed when they did an oral food challenge at the end of the study.
Almost 74 percent in the active treatment group tolerated 600 mg of peanut protein powder in a single dose (about two peanuts). That tolerated dose met the goal for the primary outcome of the study. In the placebo group, however, only about 6 percent managed to consume that amount.
Even more encouraging was the success in tolerating the highest dose of 2,000 mg of peanut protein (equivalent to about 6 to 8 peanuts), Tilles says. Sixty-one percent of treated participants could tolerate 2,000 mg compared to 2 percent on placebo.
“It was a higher magnitude of protection than generally we think of for OIT in older patients,” says Tilles.
What About Adverse Effects?
The younger children had fewer concerning adverse events, and those side effects were less likely to result in discontinuing the treatment, Tilles says.
In the study of children ages 4 to 17, anaphylactic reactions were more common in the treatment group than those on placebo. This led about 2 percent of the group to discontinue treatment. However, in the toddler study, Tilles says that while a small percentage experienced anaphylaxis, none of those patients discontinued Palforzia.
With other side effects, 9 percent of the treatment group in the older kids’ study discontinued treatment. This mostly related to symptoms like abdominal pain or nausea. In the toddler study, 5 percent stopped treatment because of GI symptoms, the main side effect for this group as well.
“Overall, the safety profile for Palforzia in toddlers is encouraging,” says Tilles. “It should help allergists engage in appropriate shared decision-making discussions with the caregivers of young children with peanut allergy.”
Toddlers’ Therapy & Lifestyle Benefits
The approval of Palforzia for toddlers presents benefits both in how it affects the youthful immune system – and for families’ lifestyles, Tilles says.
One of the challenges for older children undergoing oral immunotherapy is how the treatment impacts their daily life. For example, dosing might cause them to miss school or a sports practice.
But young toddlers in general don’t have a schedule that is as packed with activities. He says that means “it’s less of an imposition on that patient’s activities.”
Tilles stresses that the Poseidon trial and the FDA approval do not suggest Palforzia is a cure for peanut allergy. “It’s just another piece of the puzzle that’s very encouraging.”
Related Reading:
How Reliable is an Oral Food Challenge?
OIT Study Sees Maturing of Immune System Over Time
