Updates from an FDA panel’s review of Aimmune’s peanut allergy therapy – STAT

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We’re monitoring today’s meeting of the FDA advisory committee on Palforzia, a novel but controversial treatment that uses calibrated quantities of ingested peanut powder to protect people against severe peanut allergy. Check back here often for updates throughout the day.

Let’s get this FDA panel started

8 a.m.: The FDA advisory panel will be a pivotal moment for Aimmune Therapeutics (AIMT) (AIMT), the biopharma company that developed the new treatment. If approved, Palforzia (formerly AR101) will be the first protective therapy for peanut allergy and the start of what Aimmune hopes will be a family of products with blockbuster commercial potential — all designed to benefit the millions of people who suffer with life-threatening food allergies.

But the benefit demonstrated by Palforzia comes with some significant tolerability and safety concerns, which is where the crux of today’s advisory panel debate is expected to focus.

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A more onerous risk-management plan possible?

9:50 a.m.: Dr. Pamela Guerrerio, chief of the Food Allergy Research Unit at the National Institutes of Health, kicked off the substantive portion of the advisory panel with a talk on the current state of treatment for food allergy. Experts who will ultimately vote on Palforzia today quizzed Guerrerio mostly about the safety risks of an oral immunotherapy. If Palforzia is approved, the FDA will require a risk-management plan to educate people with severe peanut allergy and their families about the risk of systemic allergic reactions that can occur during the 12 months of therapy. This so-called “REMS plan” will also dictate how Palforzia is administered.

At this early stage of the advisory panel, it’s hard to judge how experts feel about Palforzia, but the questions and comments made so far suggest, perhaps, some of the voting experts lean towards a more restrictive REMS plan for the Aimmune product.

Aimmune’s presentation to the panel just started.

Aimmune’s presentation — and some panel expert feedback

12:10 p.m.: The Aimmune presentation has concluded and the advisory panel is now on a lunch break. 

Dr. A. Wesley Burks, dean of the University of North Carolina School of Medicine and a paid consultant to Aimmune, spoke in favor of Palforzia, telling the panel experts that the efficacy data from Aimmune’s clinical trial demonstrate a clinically meaningful benefit. In the real world, the sensitization to peanut protein achieved with Palforzia will lead to “peace of mind” and meaningful protection against allergic reactions from accidental exposure.

“This is what patients and families want most of all,” said Burks.

Burks also addressed the safety and tolerability issues hanging over Palforzia, calling the reported side effects and use of epinephrine during the treatment and maintenance phases of the Palforzia clinical trial “predictable and expected.” 

Aimmune also addressed the Palforzia safety concerns, acknowledging that allergic reactions did occur during the clinical trial, some of which required epinephrine to resolve, but that these reactions diminish in frequency and severity over time. 

Was Aimmune’s presentation, including Burks’ support, persuasive? It’s still hard to read the sentiment of the room. A majority of the invited experts have not commented or asked questions. It’s notable — and perhaps concerning for Aimmune – that two experts have been rather vocal and critical of the Palforzia data presented so far. 

Dr. John Kelso, an allergist at the Scripps Clinic in San Diego, said the frequency of severe or fatal allergic reactions to accidental peanut exposure in the real world are already very rare. Therefore, he’s unconvinced that the benefit reported by Palforzia is clinically meaningful, particularly given the higher rate of epinephrine required during the clinical trial. 

Dr. Erica Brittain, a statistician with the National Institutes of Health, chimed in several times to criticize the way Aimmune presented its efficacy and safety data. 

After the lunch break ends, the advisory panel will resume with a 90-minute public hearing.

Aimmune has overwhelming public support

2:30 p.m.: Over the course of 90 minutes after the lunch break, the peanut allergy community — kids, their parents and doctors — urged the panelists to vote in favor of Palforzia’s approval.  There was only a single dissenting voice.

The panel members vote

4 p.m.: Question 1: Are the available efficacy data adequate to support the use of Palforzia?

By a 7-2 margin, the panel voted “yes” supporting the approval of Palforzia. These votes are not binding, but the FDA usually follows the recommendations of its advisory panels.

The two votes against Palforzia were cast by Drs. Brittain and Kelso, the two panelists who raised concerns earlier in the day. Kelso was most stridently negative. “This is not the answer,” he said.

Dr. Soheila Maleki, a food allergy researcher at the U.S. Department of Agriculture, called the Palforzia efficacy data “impressive.” She voted to recommend approval, joining the majority of the experts on the FDA panel.

Question 2 focused on the adequacy of the proposed risk-management, or REMS, plan for Palforzia.

By an 8-1 margin, the experts agreed that the the FDA should require 1) Documentation that any patient prescribed Palforzia has a valid prescription for injectable epinephrine; 2) caregivers/patients must attest to carrying injectable epinephrine while on Palforzia; and 3 ) initial dose escalation and the first dose of each up-dosing level must be administered in a certified facility capable of treating systemic allergic reactions. 

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