Aimmune Therapeutics Inc. won the qualified backing of members of the FDA's Allergenic Products Advisory Committee (adcom) during a Friday meeting held to discuss whether or not safety and efficacy data support licensure of Palforzia (AR-101), the company's oral immunotherapy (OIT) to reduce the incidence and severity of allergic reactions to peanut.
The day's discussions featured powerful testimonials from young people and their families about the negative ways in which peanut allergies have affected their lives and the benefits they perceived in OIT. But much of the committee discussion to follow focused on parsing the meaning of clinical terms, including labels such as "systemic allergic reaction" and "anaphylaxis," so as to better elucidate the potential risks posed by intentionally exposing children to their known allergens.
One panelist expressed concerns about the value of OIT in the face of more traditional approaches, like patient education, while another worried about the very small number of black or African American patients included in the study population.
In the end, though, members of the adcom voted 7-2 in favor of there being adequate evidence supporting the use of Palforzia as a treatment to reduce the incidence and severity of allergic reactions, including anaphylaxis, after accidental exposure to peanuts in children, 4 to 17, with a confirmed diagnosis of peanut allergy, suggesting the candidate is on track to be the first peanut allergy treatment in what could be a blockbuster market.
In an 8-1 vote, the committee also supported the idea that there is adequate available safety data to support use of the drug in conjunction with a risk evaluation and mitigation strategy, or REMS, program that could require patients taking the capsule-based medicine to carry a validly prescribed epinephrine injector while on Palforzia. The program would also require that dose escalation of the medicine take place in facilities where critical allergic reactions could be treated.
The FDA accepted Aimmune's BLA for AR-101 in March 2019 and previously informed the company that completion of its review may take until January 2020. The agency granted fast track status in September 2014 and breakthrough therapy status for pediatric peanut allergy in June 2015.
Trading in company shares (NASADAQ:AIMT) was halted all day Friday during the committee's discussion. Meanwhile, Aimmune's marketing authorization application for AR-101 has been submitted to the EMA, which has validated it and begun a review.