FDA on Friday approved Aimmune Therapeutics Palforzia, the first-ever treatment for life-threatening peanut allergies.
The incidence of peanut allergies in children in the United States has increased by 21% since 2010, according to the American College of Allergy, Asthma, and Immunology. The latest estimates suggest about 2% to 5% of U.S. children have a peanut allergy.
There's a significant debate among providers about how to prevent or treat peanut allergies. Recent guidelines have recommended parents begin feeding their children peanuts at a young age to help their immune systems learn how to tolerate peanuts.
Researchers and some providers have tested a similar approach, known as oral immunotherapy, on kids who already have developed a peanut allergy. The technique involves slowly introducing children to small amounts of peanuts—but according to The Atlantic, many providers view such experiments as risky.
Palforzia is a capsule that contains small amounts of peanut powder.
For the treatment, doctors prescribe and administer a small dose of the drug, which can be mix with applesauce or another food. After the initial dose, patients must take the drug daily with doctors increasing the dose 11 times over several months until the patient is ingesting the equivalent of about one peanut. Doctors must administer each dosage increase, and if the patient tolerates the increase they may continue that dosage at home.
Researchers employed by Aimmune in 2018 published clinical trial results in the New England Journal of Medicine showing that after a one-year period, two-thirds of children taking the treatment could safely consume two peanuts in a controlled setting, compared to 4% of patients who did not take Palforzia, according to the Associated Press.
FDA on Friday approved Palforzia, making it the first drug to receive FDA approval to treat life-threatening peanut allergies. The goal of the treatment is to reduce the risk of a life-threatening allergic reaction, not to make it so that individuals with peanut allergies can regularly eat peanuts. Aimmune CMO Daniel Adelman said Palforzia is only "intended to desensitize patients to peanut protein. So while they still must practice avoidance, it can mitigate allergic reactions after accidental consumption."
The drug is approved for children ages four to 17 who have a confirmed diagnosis of peanut allergy. According to FDA, "Those who take Palforzia must continue to avoid peanuts in their diets."
Researchers assessed the effectiveness of Palforzia in a randomized, double-blind, placebo-controlled study that enrolled about 500 people who were allergic to peanuts. Researchers found 67.2% of participants who took Palforzia recipients tolerated a 600 mg dose of peanut protein after six months, compared to 4.0% of participants who received the placebo.
Researchers assessed safety using two double-blind, placebo-controlled studies in about 700 people with peanut allergies. Researchers found the most common side effects were abdominal pain, nausea vomiting, tingling in the mouth, itching (including in the mouth and ears), cough, runny nose, throat irritation and tightness, hives, wheezing and shortness of breath, and anaphylaxis. The risk of anaphylaxis is why FDA required patients to take the initial dose and the increased dosages in the presence of a doctor.
The medication will cost $890 per month, but Aimmune said it will offer an assistance program that will reduce the patient's share of the cost to as low as $20 for some patients.
Is the treatment worth the cost?
Some providers said the drug could be a "big deal" for patients who live in constant fear of making contact with peanuts.
Subhadra Siegel, chief of pediatric allergy and immunology at New York Medical College, said, "The thought of relieving that anxiety and being able to eat in a restaurant without worry. These are huge debilitating things for families with food allergies."
Two separate surveys found that allergists favor Palforzia as a treatment for patients with peanut allergies, with analysts predicting the drug will make about $52 million in 2020 and as much as $830 million in 2023.
Given the drug would be the first ever to treat life-threatening peanut allergies, Aimmune projects that Palforzia's annual sales could exceed $1 billion.
However, some experts said the new drug is simply an overpriced version of peanut flour, which can be sold as a dietary supplement at a much lower cost.
A study by the Institute for Clinical and Economic Review found that there was no sufficient evidence that using Palforzia would be more effective than avoiding peanuts or another oral immunotherapy.
But Thomas Casale—chief medical adviser at Food Allergy Research & Education (FARE), a nonprofit advocacy and lobbying group that helped create Aimmune—argued that, even though supplement manufacturers can offer cheaper versions of peanut flour, having an FDA-approved version makes the treatment safer and more accessible to patients.
Still setting aside price, the drug raises other concerns.
While researchers in an Aimmune clinical trial found that patients showed greater tolerance of peanuts in a clinical setting, outside of the laboratory they actually experienced more serious allergic events. According to The Atlantic, 14% of those receiving the peanut powder pill reported a severe allergic reaction during the study period, compared with just 3% in the placebo group. A meta-analysis published in The Lancet confirmed the risk.
Jeff Tice, a physician and health policy analyst at the University of California at San Francisco who examined the product, said, "This is just what we're trying to prevent—having to get taken to the ER … sort of outcome." He added, "The trials clearly demonstrated desensitization [to peanuts], but apparently at the cost of more harm, and no clear evidence of long-term benefit" (Hamblin, The Atlantic, 9/13/19; Johnson, Washington Post, 1/31; Loftus, Wall Street Journal, 1/31; FDA release, 1/31; Feurstein, STAT+, 2/3).