Generic name: N/A
Company: Aimmune Therapeutics
Disease: peanut allergy
2024 sales estimate: $1.75 billion
Aimmune Therapeutics’ AR101 could be the first drug that reduces allergic children’s reactions to peanut exposure. The idea is that by exposing patients to the peanut allergen in a gradual dose-escalation manner, they can be desensitized and able to tolerate higher amounts.
AR101 isn't a cure, as the company is quick to emphasize; it's meant to alleviate risks of accidental exposure. But in a 554-participant phase 3 study dubbed Palisade, more than two-thirds of patients ages 4 to 17 on AR101 tolerated 600 mg or more of peanut protein, compared with 4% in the control arm.
These patients could only tolerate a 100-mg dose of peanut protein when they first entered the trial. In real-world terms, 600 mg of protein is two peanuts or a small bite of a peanut butter sandwich.
While there’s no question about the drug’s efficacy, its safety data raised some questions. In a study called Palisade, one-fifth of nonadult AR101 patients dropped out and 12.4% withdrew because of treatment-related side effects. Aimmune executives argued that the dropout rate might not be as big a problem in the real world because doctors could adjust the dose escalation on an individual basis.
AR101's trip at the FDA hasn't been smooth, either. The government shutdown hit just after Aimmune submitted its U.S. application for approval in December, and a short-staffed FDA wasn't able to accept it for review until January. Regulators assigned the standard 12-month review, the company said—which means a decision by late January 2020. But Aimmune is in talks with the FDA about shortening the timeline, so a launch in 2019 might still happen.
Peanut allergy affects about 1 in 70 children in the U.S., and Aimmune expects to be the first in the large market. Its nearest competitor, DBV Technologies, pulled an application for its peanut allergy contender Viaskin Peanut in December but plans to refile this year. Analysts consider the DBV candidate inferior to AR101 in terms of efficacy.