The U.S. Food and Drug Administration approved Aimmune Therapeutics’ Palforzia to mitigate allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanuts. Treatment may be initiated in individuals ages 4 to 17 years with a confirmed diagnosis of peanut allergy and can continue in individuals 4 years of age and older. Patients must always avoid peanuts in the diet.
FDA Director: A Dangerous Condition
Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research reported, “Peanut allergies impact about 1 million children in the United States and only 1 out of 5 of these children will outgrow their allergy. As there is no cure, allergic individuals must strictly avoid peanut exposure to prevent severe and potentially life-threatening reactions.” He continued, “Even with strict avoidance, inadvertent exposures can and do occur. When used in conjunction with peanut avoidance, Palforzia provides an FDA—approved treatment option to help reduce risk of these allergic reactions in children with peanut allergy.”
Peanut allergy is a condition in which the body’s immune system mistakenly identifies even small amounts of peanut as harmful. Allergic reactions to peanut are unpredictable in occurrence and in how they present, with some individuals experiencing serve reactions from even trace amounts. Physical symptoms, the FDA reports, can develop within seconds of exposure and may include skin reactions (e.g. hives, redness, or swelling), digestive discomfort, or more dangerous reactions such as constriction of the throat and airways, and loss of adequate blood flow to vital organs of the body. Antihistamines and epinephrine can be used to treat allergic reactions, but severe reactions can be fatal even with appropriate, promote treatment. Palforzia cannot be used for the emergency treatment of allergic reactions, including anaphylaxis.
What is Palforzia?
Palforzia is a complex biologic drug used with a structured dosing approach that builds on a century of oral immunotherapy (OIT) research. With OIT, the specific allergenic proteins are ingested initially in very small quantities, followed by incrementally increasing amounts, resulting in the ability to mitigate allergic reactions to the allergen over time. PALFORZIA is a rigorously developed, pharmaceutical-grade OIT for peanut allergy with a well-defined allergen profile to assure that every dose, whether 0.5 mg (equivalent to 1/600th of a peanut) or 300 mg, has been prepared and analyzed for consistency.
The Biologics License Application (BLA) for PALFORZIA included efficacy and safety data from seven clinical studies, including the pivotal Phase 3 PALISADE and RAMSES clinical trials. In addition, data from the Phase 2 ARC001 study and the ARC002 open-label follow-on study were included, as well as data from ARC004, ARC008 and ARC011, which are ongoing studies.
PALFORZIA is available only through a Risk Evaluation and Mitigation Strategy (REMS). Requirements of the REMS include: the prescribing physician and patient must be enrolled in the REMS prior to initiation of treatment; the initial dose escalation and the first dose of each up-dosing level must be administered in a certified healthcare setting; epinephrine must always be immediately available to patients; and pharmacies/distributors must be certified with the REMS and dispense PALFORZIA only to certified healthcare settings or to patients who are enrolled in the REMS. Consistent with approved immunotherapies indicated to treat allergic conditions, the Prescribing Information for PALFORZIA contains a boxed warning.
Key Research Sites/Investigators
The PALISADE Phase 3 clinical trial was led by the PALISADE Group of Clinical Investigators while the Phase 3 RAMSES trial included 60 study sites. The Phase 2 ARC001 study included the following sites: Arkansas Children’s Hospital; UC San Diego; Boston Children’s Hospital; Mount Sinai Medical Center; University of North Carolina; Cincinnati Children’s Hospital Medical Center; The Children’s Hospital of Philadelphia and Children’s Medical Center, Dallas. The Phase 3 ARC004 study follow-on study is complete. Study ARC008 is to assess AR101’s safety and tolerability over and extended dosing period. Clinical investigators are not listed. Ellen Ashley, Study Chair, is Director of Clinical Operations.
For dosing and other relevant safety information, follow the link to the FDA’s website.
Who is Aimmune Therapeutics?
Aimmune Therapeutics, Inc. is a biopharmaceutical company that aspires to become the global leader in developing curative therapies and solutions for patients with food allergies. With a mission to improve the lives of people with food allergies, Aimmune is developing and commercializing oral treatments for potentially life-threatening food allergies. The Company’s Characterized Oral Desensitization ImmunoTherapy (CODIT™) approach is intended to provide meaningful levels of protection against allergic reactions resulting from accidental exposure to food allergens by desensitizing patients with defined, precise amounts of key allergens. Aimmune has one FDA-approved medicine for peanut allergy and other investigational therapies in development to treat other food allergies.
Business & Finance
Priced at $31.05 with a market capitalization of $1.97 billion, the company reports losses most recently in the -$237 million range. They have accumulated significant deficits losing $210.7 million in 2018; $131.3 million in 2017; $80.8 million in 2016 and so on. They possess about $195 million cash and employ about 300-350.
Strategy Moving Forward
Their declared strategy in investor disclosures included the completion of development and securing of approval of AR101—this major milestone was achieved with the FDA’s recent news. They will now seek to monetize the approval of (AR101/Palforzia) with the growth of a specialty sales force targeting practicing allergists in the United States and allergy-focused clinicians in major European markets. This will help them incur even greater expenses. They hope to drive sufficient revenues to cover costs at some point and of course establish commercial infrastructure for other prospective product candidates.