First peanut allergy drug approved by FDA

The first drug to prevent potentially life-threatening allergic reactions to peanuts was approved Friday, providing children and families who live with the fear of accidental exposures a long-awaited treatment option.

The therapy, Palforzia, isn't a cure and comes with significant risks of triggering the very reactions it is supposed to quell. But for families and children who have re-engineered their routines to minimize potential exposures - changing how and whether they travel, eat out, socialize and feel safe in their daily lives - it could offer an important layer of protection and relief.

Palforzia is seen as an important test case for a new generation of therapies expected to transform how a rising epidemic of food allergies are treated. Doctors who have had few tools other than counseling their patients to assiduously avoid peanuts expect that other drugs will follow - additional drugs for peanut allergy, as well as egg and tree nut allergies. Aimmune Therapeutics, which makes Palforzia, has several other food allergy treatments in its pipeline.

"Even with strict avoidance, inadvertent exposures can and do occur. When used in conjunction with peanut avoidance, Palforzia provides an FDA-approved treatment option to help reduce the risk of these allergic reactions," said Peter Marks, director of the Food and Drug Administration's Center for Biologics Evaluation and Research.

Allergists have emphasized that the drug will not be appropriate for every family. It is approved for children ages 4 to 17 and requires children to eat increasing doses of peanut protein to gradually desensitize their allergies. That means patients must intentionally expose themselves to the very substance that causes trigger a life-threatening reaction under the regimen, called oral immunotherapy. Due to the risks, patients must take some doses under a doctor's supervision. The drug can cause severe allergic reactions and require epinephrine injections. It's unclear how long patients will have to stay on the drug.

Palforzia, which will carry a list price of $890 per month, could also reignite discussions about high drug prices - although the company will offer an assistance program to drop patient's share of the cost to as low as $20. Some physicians have already used this oral immunotherapy approach to desensitize people to peanut allergies using commercially available, inexpensive peanut flour. Proponents of Palforzia argue that having a drug available could standardize the treatment, provide a way for insurance to cover it and also ensure that there isn't variability in the amount of allergen in different batches of peanut flour.

But a study by the Institute for Clinical and Economic Review, which evaluates whether drugs are worth the price, found that there wasn't sufficient evidence that Palforzia was better than strictly avoiding peanuts or than current immunotherapy that uses peanut flour.

The therapy has been life-changing for Giuliana Ortega, 8 of Hughesville, Maryland. Her mother, Alicia, vividly recalls when Giuliana ate a peanut butter cracker when she was two and a half years old. She was vomiting white foam by the time a steroid finally started to combat the reaction. Later, in pre-kindergarden, a friend who had eaten a peanut butter sandwich hours earlier handed her his kazoo. After she blew into it, she had a severe reaction.

Giuliana has been taking Palforzia through a clinical trial since she was four and said that the therapy has changed her life. Now, she can choose whether to sit at the peanut-free table at lunch or with the other kids, take a flight to Florida and even hug family members without having to ask them if they've eaten peanuts recently.

"I've gone on a vacation, can sit at a normal table with the normal kids when I want to, go to the theater without wiping the seat down, can go to a hotel safely, can fly on a plane now," Giuliana said. She said the first time that her mom told her she could sit at the lunch table where kids might be eating peanut butter sandwiches or other food with peanuts she was scared, but that she felt fine.

But Giuliana's experience also demonstrates how much commitment the drug will take, and that it may not be for everyone. As part of the treatment, she went to the hospital every other Friday to be bumped up to a higher level of the drug under medical supervision. The medicine caused some allergic reactions and stomach cramps, and her parents had to be careful that their daughter, who loves to dance, stayed calm and still after she took the drug. If she ran up the stairs, for example, it could cause her to metabolize the drug too fast and trigger a reaction.

Under the standards set in the trial, Giuliana didn't have a clear cut success - she was not able to eat the equivalent of 2 peanuts without having a reaction. But her mother, Alicia, said that the drug has been critical in giving the family a new level of confidence.

They have begun to eat at new places - including a special breakfast each week when her dad would bring back doughnuts. One day last summer, after Giuliana ate the same doughnut she'd had safely many times, Alicia noticed that her daughter was getting a puffy, swollen look around her eyes that usually preceded a reaction. Alicia called the shop and found out that a new doughnut of the month had just been introduced that contained peanut, possibly cross-contaminating the one her daughter was eating. But the miraculous thing was that Giuliana took a Claritin, and her reaction subsided.

"That's kind of what we wanted, just a normal life," Alicia Ortega said. They're still vigilant about peanuts, but it has been life-changing. "It's given us a confidence that we would never have had."