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In a historic vote Friday, an advisory group recommended Aimmune Peanut Allergy Immunotherapy for FDA approval. The treatment, which desensitises patients by giving them increasingly important doses of peanut protein, could change the deal in the area of food allergies, where the most common treatment is to avoid it.
The panel voted 7-2 to approve the treatment, known as AR101 and marketed as Palforzia's approved, to "reduce the incidence and severity of allergic reactions, including anaphylaxis, after accidental exposure to peanut "in children and adolescents with diagnosis of peanut allergy.
The panel voted 8-1 in favor of a safety plan called REMS, proposed by the FDA, to assess risks and mitigation strategies, to support the use of Palforzia in children from 4 to 17 years old. This plan provides that while Palforzia's prescription patient has valid prescription for injectable epinephrine, caregivers / patients attest that they carry injectable epinephrine during treatment with Palforzia, as well as the drug. increase in initial dosage and that the first dose of each level of over-dosage should be administered at a certified site capable of treating systemic allergic reactions. Although the panel's vote is not binding, the FDA generally tends to follow the committee's recommendations.
Aimmune opened in 2012 to develop approved treatments for food allergies. It is a group of parents, advocates, researchers and FDA members frustrated by the lack of regulated treatments for life-threatening allergies.
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The idea behind Palforzia is not new. Doctors use immunotherapy to treat other types of allergies, such as those caused by bee venom, ragweed and pollen. It involves injecting patients with ever larger doses of what they are allergic to, hoping to reach a point where their immune system can tolerate it.
"What I would say about Palforzia, once approved, is exactly the same as other immunotherapies, start at a low dose, slowly increase, charge the immune system the same way you do. try to counter an immune response, "Jayson Dallas, CEO of Aimmune, told Fierce Biotech.
The difference is the delivery.
"In the case of a food allergy, it's a bit more risky to do something like this with injections, so Aimmune did it as an oral immunotherapy," said Thomas Casale, MD, the Chief Medical Advisor of FARE (Food Allergy Research & Education), says FierceBiotech. FARE provided early funding for Aimmune and Casale was the lead investigator of the Palforzia Phase 3 trial in Tampa Bay, Florida.
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The BLA package for Palforzia includes data from two Phase 3 studies and their follow-up studies. The PALISADE trial, which lasted a full year, tested placebo treatment in 555 patients aged 4 to 55 years. The vast majority of them were children and teenagers, the age group for which Aimmune was seeking approval.
At the beginning of the trial, patients could not tolerate 100 mg or less of peanut protein – it is a fraction of a single peanut weighing about 300 mg. Patients received a higher dose of treatment every two weeks until reaching a maintenance dose of 300 mg. After one year of treatment, 67% of patients in the intent-to-treat population could tolerate 600mg of peanut protein, approximately two peanuts or a small bite of a peanut butter sandwich, compared to 4% of patients receiving a placebo.
"In the real world, we try to protect people, it's pretty much a peanut," said FierceBiotech, CEO of Aimmune. "About 125 mg is the median dose that results in accidental exposure to a life-threatening systemic allergic reaction when people try to avoid peanuts in the real world."
Two panellists, Erica Brittain, a statistician at the National Institutes of Health, and Soheila Maleki, a food allergy researcher with the US Department of Agriculture, raised the issue that Palforzia could be a lifelong treatment.
"[The fact that] we do not have long-term follow-up tapping gives me a break, "Brittain said.
Aimmune works there. More than 90% of patients who completed PALISADE were included in the follow-up study.
"Our goal is to continue to treat them until they begin a remission of their allergy and if they stop the treatment they are no longer allergic," Dallas said.
If immunotherapy against grass pollen or bee venom is an indication, one year at Palforzia is far from enough to wean patients, added Steve Tilles, director of medical affairs at Aimmune.
"With grass pollen allergy injections or bee venom immunotherapy, if we stop treatment after the second year, allergy will re-accumulate and the patient will remain as sensitive as it was before the beginning, "said Tilles. "The magic number with these two types of immunotherapy is three years."
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One of the two dissident jury members, John Kelso, MD, an allergist at the Scripps Clinic in San Diego, has echoed concerns over safety data. In the Phase 3 study, patients taking Palforzia had a higher rate of allergic reactions, use of epinephrine, and discontinuation than the placebo group: 89 , 1% versus 57.5%.
"The question is about reducing reactions to accidental exposures [which are] not directly evaluated and would be very difficult to evaluate directly, "said Kelso." I understand the reason for substituting this substitute to pass the challenge of food, but I think because the treatment itself represents a [food] Each time a dose is administered and the end result being that patients on treatment are twice as likely to have a reaction requiring epinephrine, the efficacy of the treatment has not been demonstrated. It can be demonstrated the day the food challenge was made. But the rest of the time, patients face daily challenges in the form of a dose of medication that does not decrease, but increases the risk of reaction. "
However, Casale and Dallas pointed out that these reactions occurred under the supervision of doctors and parents, and that it was taught to patients and their caregivers to recognize and quickly treat the symptoms of a reaction. allergic.
"[It] is very different in my mind than having an allergic reaction when you do not expect it, if your child is at a party and you are not there with them and that they do not have epinephrine to treat them if they have an accidental exposure, "Casale said.
In addition, if Palforzia is approved, allergists can take steps to reduce the risk of reaction to treatment, "said Dallas.
The two-week start-up schedule should be considered a "speed limit," he said.
"In clinical practice, patients can take the necessary time at each intermediate dose to reach 300 mg. This is exactly what allergists do today in the development of immunotherapy for grass and pollen allergies, "he said.
Doctors can also use an antihistamine alongside Palforzia to "lessen" the allergic reactions and gastrointestinal disorders that may occur.
Overall, it's "probably not a perfect therapy," Casale said. "But it's a first step and I think that's what generates a lot of enthusiasm. Currently, very few doctors offer this type of therapy because we do not know exactly how to do it. Approval of this product will encourage more allergists to use this drug. [oral immunotherapy] in practice, it is hoped that patients will have access to it and will be able to make an informed decision if it suits them, if they prefer to wait for something else or if they prefer to simply avoid the risks. "
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