Peanuts are a uniquely dangerous legume. A small fragment of one can kill an allergic person. Even if that person is resuscitated, it’s possible to have permanent brain damage. These severe allergic reactions are relatively rare but difficult to predict. One time, you accidentally eat a bit of peanut and get a little itchy; the next time, your airways close up and someone has to jam a needle full of adrenaline into your leg just so you can take a breath.
A controversial treatment was approved by the Food and Drug Administration on September 13th after a review with the pharmaceutical company Aimmune and its advocates. Allergenic Products Advisory Committee (APAC) convened by the U.S. Food and Drug Administration (FDA) voted to support the use of AR101 (proposed trade name Palforzia™).
“We are very pleased that APAC voted in favor of Palforzia. This is an important day for the children, teens and their families who live with the profound daily impact of peanut allergy. We look forward to continuing to work with the FDA as we move towards a potential approval of Palforzia,” said Jayson Dallas, M.D., President and Chief Executive Officer of Aimmune. “We are immensely grateful to the entire food allergy community for helping us bring Palforzia one step closer to becoming the first FDA-approved treatment for any food allergy.”
There is currently no “treatment” for a peanut allergy. As it is, patients are told to avoid peanuts. They are prescribed a syringe full of epinephrine (trade name: EpiPen) and taught to inject themselves if needed. Despite much advancement in medical science and technology over the decades, nothing has given families peace of mind that the allergy itself could be treated, or at least tempered. Until now. The new approach involves trying to reprogram the immune system by giving a person … peanuts.
Immunotherapy is common practice in the form of allergy shots for things such as pollen: You inject bits of the protein and watch as the immune system gets more tolerant and the person becomes less wheezy. But oral immunotherapy with peanuts is considered experimental, and no professional organization recommends that parents give any peanuts to an allergic person.
“The problem was that this wasn’t exactly a product that a typical drug company would make,” says Brian Vickery, an allergist involved in the proof-of-concept studies. Nor was the product a food, exactly. “And there was nothing to be licensed—it was peanut flour, you know?”
Food Allergy Research & Education, or FARE, a nonprofit advocacy and lobbying group that’s partly funded by the pharmaceutical industry found a company that would become known as Aimmune. Though Aimmune was technically a pharmaceutical company, its work focused on making food-based therapies. These are regulated by the FDA as drugs, but under the new and growing category of “biologics”—drugs that come from living organisms, such as vaccines made from bacterial proteins or insulin made from the human hormone. In Aimmune’s case, the biologic would be peanut flour placed in a capsule. The company went public in 2015 after raising $160 million in venture-capital funding.
Based on their single, year-long trial, Aimmune is now petitioning the FDA to approve its powder as a drug. Instead of “peanut flour,” Aimmune calls the drug Palforzia. It does not promise to give people an ability to eat peanuts—only to potentially protect a person in the case of a small, accidental ingestion. Analysts have put the cost at $4,200 a year. The drug would have to be taken indefinitely.
“There’s tremendous demand, because patients feel helpless and terrified,” says Jeff Tice, a physician and health-policy analyst at the University of California at San Francisco. “The insurance companies are getting ready for a large financial impact because this will get taken up quickly.”
Desensitizing a person to small amounts of peanut doesn’t mean Palforzia extends people’s lives. It doesn’t mean the drug keeps them out of the hospital or prevents serious allergic attacks. In other words, based on what is currently known, the billion-dollar peanut powder is likely to have a net effect of increasing severe allergic reactions to peanuts.
Vickery, who is no longer in the full-time employ of Aimmune, admits that “for an expensive therapy, we don’t know if [Palforzia] even does what it’s supposed to do.” But he sees the implications of this approval as far bigger than peanut powder. “If a start-up can do a trial of 1,000 patients and actually get something to market as the first approved drug for peanut allergy,” he says, investors would flock to the area.
Call to Action: Subscribe to TrialSite News to read follow-up information on the FDA review of Palforzia.Source: The Atlantic