- Aimmune Therapeutics' peanut allergy treatment on Friday passed a crucial test in its path toward approval, as a panel of scientific experts convened by the Food and Drug Administration voted in support of the drug's safety and efficacy.
- After hearing from experts, physicians, people with peanut allergies and company officials, the FDA's advisory committee voted 7 to 2 in favor of the drug's effectiveness and 8 to 1 on safety. But some experts raised concerns about higher rates of epinephrine use, as well as systemic allergic reactions, among those treated with drug compared to those on placebo.
- The FDA is not required to follow the advice of its committees, but they are seen as influential in guiding regulatory decisions. Aimmune expects a decision on U.S. approval by late January 2020.
AR101's road to approval became clearer Friday. Yet the committee's review also demonstrated the significant challenges that will lie ahead for the peanut immunotherapy if cleared for sale.
By targeting food allergies, Aimmune looks to enter a market with few treatment options. A 90-minute session for public comment featured parents, children and patient advocates describing the daily fear and anxiety that peanut allergies bring. Standard treatment typically involves avoidance and epinephrine to treat allergic reactions.
"Avoidance alone is simply not enough," said Charmayne Anderson, advocacy director for the Allergy & Asthma Network, a nonprofit that has taken funding from Aimmune.
Aimmune's drug, known as AR101 in clinical testing, is an oral immunotherapy consisting of peanut powder taken daily along with food. Clinical trials showed AR101 outperformed placebo in improving study participants' ability to consumer peanuts at food "challenges" presented on exiting the trial.
But some members of the FDA's voting group held reservations about the drug's risk-benefit profile.
"It is not clear to me that this treatment is effective," said John Kelso, a California-based allergist and the sole committee member to vote against AR101 on both efficacy and safety.
Kelso emphasized that epinephrine was used about twice as frequently among those who took AR101 instead of placebo. In his view, using an exit food challenge as the study's primary endpoint prevented the committee from directly answering whether the drug can reduce the incidence and severity of allergic reactions.
The panel also discussed a Risk Evaluation and Mitigation Strategies (REMS) plan that Aimmune and regulators are finalizing. The company shared some of the proposed label language, which includes recommending people who take the drug avoid hot showers or exercise in the hours after dosing.
FDA staff said the agency would require people have documentation for an epinephrine prescription, attest to carry epinephrine and limit dose escalation to only certified facilities.
"This may not be a perfect solution, but it is the first solution which I'm sure will lead to many others," David Anmuth, a private allergist in Fairfax, Virginia, said during the public comment period. "They would rather take their chances in a controlled environment than take their chances each day."
Aimmune plans to sell AR101 as Palforzia and is considering a list price between $3,000 and $20,000 if the drug's approved, according to a recent company presentation.
"This product has met that standard and deserves a chance to be seen in the marketplace and see where it floats," said committee member Gailen Marshall, an allergist at the University of Mississippi Medical Center.