Fear Factor: Palforzia

Living with peanut allergy can be like living in an earthquake zone. Sufferers might go through life enduring tremors in the form of a tingling sensation around the mouth or hives, but the life-threatening chance of “the big one” always looms. Not knowing if or when it will occur can leave patients and their families in a constant state of anxiety. The Jan. 31, 2020, FDA approval of Aimmune Therapeutic’s Palforzia® [Peanut (arachis hypogaea) Allergen Powder-dnfp] has helped relieve some of that stress.

“Even if you don’t have children or family members or are yourself affected by food allergies…the impact of these things on people’s daily lives [can be great],” says Andrew Oxtoby, president and CEO of Aimmune. “It’s completely disruptive in terms of whether or not [families] decide to eat at restaurants or go on vacation. It is something beyond the clinical aspect of a potential inflammatory reaction. It’s not knowing day to day whether your child is going to be exposed to something that may potentially kill them.”

Aimmune’s new product, Palforzia, is an oral immunotherapy treatment for peanut allergy that gradually exposes patients to increasing amounts of the allergen. Over time, patients are able to tolerate a greater amount, and their immune system is essentially reprogrammed until they reach a maintenance phase.

“We’re not looking to have it so that people can dig into peanut butter sandwiches,” says Oxtoby. “What we’re trying to do is make sure that in the event that they are exposed to some amounts of peanut, either inadvertently by eating another cross-contaminated food or by being exposed to it, the chance of them having a severe reaction is greatly reduced.”

The treatment is approved for children ages 4 to 17, and those who have experienced a serious allergic reaction seem to be highly motivated to take it. “Palforzia can instill confidence that there is an FDA-approved option, which is covered by insurance programs, that families can discuss with their physician,” says Oxtoby. “It is well-researched, well-studied, and has a well-characterized manufacturing process. It provides them the chance to take back control of their lives. It’s about empowerment.”

The prevalence of peanut allergy among children has risen in recent years. In fact, a 2013 report by the Centers for Disease Control and Prevention estimated that between 1997 and 1999, food allergies affected about 3.4% of American children. By 2011, that number reached 5.1%. According to the organization Food Allergy Research & Education (FARE), results from a 2015–2016 survey indicate that 5.6 million children, or nearly 8%, have food allergies. That’s about two children in every classroom.

Introducing children to foods earlier in life can be one way of addressing allergies. Oxtoby hopes that therapies such as Palforzia will start having an impact and ultimately cause a decrease in the incidence of allergic reactions.

Oral immunotherapy has been around for many decades in various conditions, including food allergy. There are centers around the US that offer similar desensitization treatments for a variety of food allergies.

However, the difference with Palforzia is that patients now have the knowledge that the drug has been rigorously studied through randomized controlled trials and approved by FDA. Every dose has the same allergenic profile and is precisely weighed so families know that their child is getting exactly what is prescribed.

“In some cases, the very first dose is the equivalent of one-six-hundredths of a peanut,” says Oxtoby. “While some individual clinics may have been doing this in an off-label fashion for years, precisely measuring out one-six-hundredth of a peanut consistently, accurately weighing the amount of peanut, and having the right and consistent allergenic profile through the up-dosing phase is a difficult process, as you can imagine. We’ve been able to bring that through the formal manufacturing process and clinical trial rigor that we have.”

While some food allergies naturally alleviate over time, peanut allergy is persistent—80% of children do not outgrow it. In contrast, egg and milk allergies only last into adulthood about 20% of the time. This is one reason Aimmune chose to address peanut allergy first. However, the company currently has a Phase II program in egg allergy, as well as a multi-nut allergy product.

Launching Palforzia in the face of the COVID-19 pandemic wasn’t easy. “I certainly would never recommend anybody launch a new product in the midst of a global pandemic,” says Oxtoby. “It’s not advisable. But nobody could have predicted this. We got approved on Jan. 31 and set about our launch as we had originally planned. We had our account managers trained and talking to allergists during February, getting them certified in the [FDA-required] Risk Evaluation and Mitigation Strategy [REMS] program to be able to administer the product.”

Oxtoby recalls the first child was given Palforzia in Atlanta on March 13, 2020—the exact day the US president declared a national state of emergency. The most acute effect was that many allergy practices closed their doors. As they gradually reopened, their attention was not on adopting new immunotherapies, however. Many were simply trying to stay in business, provide care to existing patients, and address more common issues such as environmental allergies.

Aimmune was unable to move forward with some of the normal launch activities it had planned, including immersive in-person training workshops scheduled around the country. In addition to rethinking launch strategies, the company had to face issues with the treatment itself.

“We didn’t want to appear tone deaf, to ignore the fact that our treatment requires patients to go into the physician’s office for 11 up-doses during a six-month period,” says Oxtoby. “That’s something that in a normal environment people understand and can sign up for and plan for. In an environment where people were reluctant to leave their homes, that’s not ideal. So, we put a lot of things on hold until this year, and we’ve just really started to launch this drug in the way that we always intended to.”

Another aspect of COVID that affected Palforzia’s uptake were modified school schedules that often eliminated lunchtimes. Many parents weren’t in a rush to adopt the therapy because they were able to closely monitor their children’s diets at home. Even though they understood the long-term benefit of Palforzia, there was no immediate need to begin treatment during lockdown. In fact, many patients have signed up for the program but are waiting to start the product until school resumes on a more normal schedule.

COVID wasn’t the only disruptor for Aimmune during 2020. Oxtoby, who was chief commercial officer at the time of launch, transitioned to his current role of president and CEO in October 2020, just when the company was being acquired by Nestlé Health Science and right before Palforzia’s EU approval in December. Managing such change reinforced the value of frequent communication for Oxtoby. “With people working virtually, you have to think about how to make sure they feel like they’re still part of the company and the community, and focused on the mission,” he says.

Oxtoby understands that not every drug is right for every patient, but Palforzia marks a major step forward for the food allergy community. “We’re the first and only approved treatment for any food allergy,” he says. “There are over 1.6 million children within our label with peanut allergy in the US. There’s an awful lot of children out there that are hopefully going to benefit from this.”

Read the profiles of all 2021 product launch selections here.

Elaine Quilici is a Senior Editor for Pharm Exec. She can be reached at equilici@mjhlifesciences.com.