The FDA has just added a fresh boost to Acadia Pharmaceuticals, naming its late-stage Parkinson's drug pimavanserin a "breakthrough" therapy deserving rapid regulatory response as it nears the finish line with a closely watched marketing application.
This is just the latest in a string of bullish moves for San Diego-based Acadia. A little more than a year ago the biotech reported that the FDA had waved off the need for a confirmatory study of the drug, setting the stage for an application before the end of this year.
Back in early 2013 investigators noted that in addition to hitting its primary endpoint in a Phase III trial--demonstrating highly significant antipsychotic efficacy on a reduced 9-item SAPS-PD scale--pimavanserin also scored well on key secondary endpoints. The drug arm was significantly less likely to suffer from hallucinations and delusions than the placebo arm and was twice as likely to rate as "very much improved" or "much improved."
Shares of Acadia were up about 12% in early trading on Tuesday. The biotech plans to market the drug as Nuplazid and currently holds full global rights to the therapy, which could be the first such drug of its kind to tackle psychosis related to Parkinson's. The drug is a small molecule that is a selective serotonin inverse agonist targeting 5-HT2A receptors. And it's also in Phase II studies for schizophrenia as well as Alzheimer's, which could add significantly to its long-term value.
The breakthrough drug designation was rolled out early last year as the FDA promised to more rapidly address cutting-edge therapies in the pipeline. Essentially, regulators promise to keep an open door for this select group of drugs, greasing the regulatory tracks and possibly reducing the amount of time it takes to get a drug to the market.
"The hallucinations and delusions in Parkinson's disease psychosis are devastating to patients and contribute to a dramatic rise in caregiver burden," said Joyce Oberdorf, president and chief executive officer of the National Parkinson Foundation. "Parkinson's disease psychosis is a leading cause of nursing home placement for Parkinson's patients and, with no FDA-approved therapy for this serious condition, there is a great unmet medical need for an effective, safe, and well-tolerated treatment option for patients."
- here's the release