A panel of experts convened by the Food and Drug Administration voted in support of the agency approving a drug to treat patients with peanut allergy.
The FDA’s Allergenic Products Advisory Committee voted 7-2 Friday that available efficacy data were adequate to support the use of Aimmune Therapeutics’ Palforzia, also known as AR101, among patients aged 4 to 17. It also voted 8-1 that the safety profile supported the drug’s use, provided it come with a Risk Evaluation and Mitigation Strategy, or REMS.
The company suspended trading of its shares on the Nasdaq Friday morning ahead of the AdCom meeting.
A spokesperson for the company wrote in an email that there is not a specific action date for the FDA to reach a decision, but the expectation is that it will decide in January 2020, based on the 12-month approval timeline. The FDA is not required to follow AdCom recommendations, but usually does.
“This recommendation recognizes the urgent need for patients to have a treatment option for their potentially life-threatening allergy,” said Aimmune CEO Jayson Dallas, in an emailed statement from the company. “There are over 1.6 million children and teens in the U.S., many of whom are anxiously waiting for an FDA-approved medicine that will provide them with an option beyond avoidance alone.”
In addition to the REMS, Marion Gruber, director of the FDA’s Office of Vaccines Research and Review at the Center for Biologics Evaluation and Research, said the drug’s label would carry a black-box warning. The REMS would require that any patient prescribed Palforzia had a valid prescription for epinephrine, that caregivers attest to carrying injectable epinephrine and that initial dose escalation and the first dose of each up-dosing level be administered in a certified facility capable of treating systemic allergic reactions.
A higher rate of systemic allergic reactions – including anaphylaxis – was observed among patients in the study group compared with those who received placebo. That led two panelists – Dr. John Kelso, an allergy and immunology physician at San Diego’s Scripps Clinic, and Hospital of the University of Pennsylvania Professor Dr. Andrea Apter – to vote no on the first question, about efficacy. However, Apter was among the ayes on the second question, about safety, while Kelso maintained his no vote.
Other physicians interviewed previously about Palforzia had a more favorable view, stating that the safety profile would improve over time and was consistent with other oral desensitization regimens.
Still, Kelso expressed the view during the meeting that education rather than oral immunotherapy could address “a good proportion” of the concerns heard from peanut allergy patients during the meeting’s comment period. “Anaphylaxis requires ingestion, so for the overwhelming majority of patients with peanut allergy, sitting next to another child eating a peanut butter and jelly sandwich and getting some on the skin will not cause a serious reaction,” Kelso said.
Along with other food allergies, peanut allergy has been on the rise in recent years. Among U.S. children, the incidence of peanut or tree nut allergy appears to have tripled between 1997 and 2008, according to Food Allergy Research and Education, an advocacy group.
Apter also said, however, that there was not sufficient diversity in the Phase III study, with very few African-American patients included. “There may not be a difference, but there may be too,” she said. Another panelist, Inglewood, California, private practice physician Dr. Randy Hawkins, echoed that view. “I do want more diversity in this study,” he said, adding that he wanted to see more African, Asian and – especially relevant to his Southern California practice – Hispanic patients.
According to a briefing document posted online earlier this week, white patients made up 78.5 percent of the trial’s 372-patient treatment arm and 78.2 percent of its 124-patient placebo arm. By contrast, African-American patients made up 1.6 percent and 2.4 percent, respectively, for a total of only nine patients between the arms. A respective 11 percent and 6.5 percent of patients were Asian, and Hispanic or Latino patients made up 7.8 percent and 12.1 percent. Only one patient, in the treatment arm, was Native American or Alaska Native, and only one was Native Hawaiian or Pacific Islander, also in the treatment arm.
Indeed, clinical trial diversity is a topic of growing importance among many experts, given that results with insufficient diversity may not reflect the general population. However, it is also a complex issue to address.
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