Acadia expresses dismay over FDA rejection

April 5, 2021

This week, the FDA slapped down Acadia Pharmaceuticals’ bid to win an expanded indication for its anti-psychosis drug.

Called pimavanserin (Nuplazid), the drug is the only FDA-approved treatment for hallucinations and delusions associated with Parkinson’s disease psychosis. The new approval hopes were pinned on expanding its use to patients with dementia-related psychosis (DRP).

Acadia has a lot riding on Nuplazid. Last year, the drug accounted for all of the company’s $442 million in sales — a 30 percent increase over 2019. After positive top line results from a study of the drug on dementia patients in December 2019, an SVB Leerink analyst predicted that an expanded label could propel it to blockbuster level sales.

Yet, in March, Acadia reported that the FDA said there were deficiencies in the company’s application, causing analysts to speculate that approval could be delayed. Ultimately, the FDA issued a complete response letter this week. Despite meeting the primary and secondary endpoints of its study on patients with DRP, the FDA said that the company had failed to include enough patients in its study with certain subtypes of dementia. 

In response, the company’s CEO said on an investor call on Monday that the rejection was “disappointing” and “not consistent” with the Acadia’s previous agreements with the FDA on the trial’s design. 

"Acadia stands behind the robustly positive results from the pivotal Phase 3 HARMONY study and the prospectively agreed trial design and criteria for establishing efficacy in DRP. Over the entire course of the review, the Division did not raise any concerns regarding the agreed upon study design, including the issues raised in the CRL," Steve Davis, CEO of Acadia, said in a press release. 

The company now plans to schedule a Type A meeting with the FDA to discuss a path forward for the drug’s expanded approval.