Another FDA 'No' For Acacia Over Unresolved Facility Deficiencies
Executive Summary
Confusion reigns at the UK firm as a second attempt to get approval for its nausea and vomiting treatment Barhemsys floundered following an insufficient resolution of manufacturing deficiencies at the supplier of the drug.
You may also be interested in...
Keeping Track: US FDA Approves Pfizer’s Vyndaqel, Jacobus’ Ruzurgi, But Nixes Acacia’s Barhemsys Again
The latest drug development news and highlights from our US FDA Performance Tracker.
PTC Confident About Commercial Prospects For Gene Therapy Upstaza
PTC CEO Stuart Peltz tells Scrip that cost watchdogs are likely to look favorably on the firm’s transformational gene therapy for a distressing rare brain disorder in children who could not even lift their heads before treatment.
Orphazyme’s Sorry Story Ends In Fire Sale
While Orphazyme's attempts to get approval for arimoclomol failed miserably, new owner KemPharm is confident it can get the oral heat shock protein amplifier across the regulatory finishing line for Neimann-Pick disease type C.