New eBook: The role of real-world evidence in FDA approvals. Download now.

FDA Decision Alerts July 23, 2020

CDER-Approved NDA for ONGENTYS® (opicapone)

Nick Honig and Shannon Reynolds
Contributing writers, Aetion

On April 24, 2020, the FDA approved Neurocrine’s ONGENTYS® (opicapone) “as an adjunctive treatment to levodopa/carbidopa in patients with Parkinson’s disease (PD) experiencing ‘off’ episodes.”

Key findings from the FDA’s Medical Review:

The results of two controlled, double-blinded, randomized controlled trials (RCTs) provided primary evidence of the effectiveness of opicapone to reduce “off” time (i.e., increase “on” time with good function) without any “new, novel, or unexpected adverse events.” The applicant also submitted postmarket data to support the efficacy and safety of opicapone.

Purpose
At the time of FDA approval, opicapone was authorized for use in 32 countries. The applicant submitted postmarket data to support efficacy and safety.

What was done
The applicant submitted yearly Development Safety Update Reports (DSURs) to the FDA as part of its application for opicapone. Due to the cut-off date for the final DSUR, the applicant submitted postmarket data from Germany, Italy, Portugal, Spain, and the U.K. According to the applicant’s estimate, exposure to opicapone totaled 35,013 patient years.

Outcome
Efficacy: The FDA found the postmarket data provided “no additional information on efficacy.” The FDA found the two RCTs sufficient to support a determination of benefit for opicapone. 

Safety: The DSUR provided information on 722 “spontaneously reported” adverse events, 116 of which were classified as serious. The DSUR also reported 123 instances where treatment with opicapone was discontinued. The FDA stated the postmarketing data “has not revealed any new or novel serious or unexpected adverse drug reactions.”


FDA Decision Alerts

Subscribe to our newsletter for insights and updates on research and applications for real-world evidence in life sciences and value-based health care.

eBook

The role of real-world evidence in FDA approvals

View now
arrowcalendarchevronclosecollapsecountdownfacebookflickrinstagramlinelinkedinlocationpinsearchsocialtwitteryoutube