How often does the FDA tell a drug developer to dispense with a requisite, second late-stage trial—and to proceed with filing a new drug application?
Shares of San Diego’s Acadia Pharmaceuticals (NASDAQ: ACAD) appreciated by two-thirds today, leaping from yesterday’s close of $7.97 a share to more than $13.30 in late mid-day trading on the Nasdaq market—after Acadia said the FDA agreed the company had enough compelling data for pimavanserin, its lead drug candidate proposed for treating for Parkinson’s disease psychosis.
The FDA usually requires at least two successful late-stage trials, but will accept a single trial under certain circumstances, such as a drug that satisfies an unmet medical need.
In today’s statement, Acadia says that after meeting recently with FDA regulators, the company no longer plans to conduct the second Phase 3 study that was planned as a confirmatory trial. That trial had been scheduled to begin this month.
In a conference call this morning with analysts and shareholders, Acadia Chief Medical Officer Roger G.M. Mills, said the company plans to conduct standard development studies and collect related data needed to submit an NDA, now targeted for the end of next year.
Mills said a meeting the company held with FDA regulators earlier this week was intended to show how the company’s pivotal study met the agency’s criteria for using a single study. According to a transcript prepared by Seeking Alpha, Mills said, “The pivotal -020 Study was a large, multicenter study with statistically very persuasive results that were consistent across study subsets. The -020 Study also assessed multiple endpoints involving different events including not only antipsychotic efficacy, but also positive effects on nighttime sleep, daytime wakefulness and on caregiver burden.”
In addition to the strength of results in the study, Mills said Parkinson’s psychosis is a serious unmet medical need without any approved treatment option. About 40 percent of patients with advanced Parkinson’s suffer from hallucinations and manifestations of psychosis, according to the National Institutes of Health. Parkinsons occurs in about 0.3 percent of the population of industrialized countries, affecting between eight and 18 people out of every 100,000.