Reuters logo
Horizon Pharma drug fails key study in rare neuromuscular disease
December 8, 2016 / 12:19 PM / a year ago

Horizon Pharma drug fails key study in rare neuromuscular disease

Dec 8 (Reuters) - Horizon Pharma Plc said its drug, Actimmune, failed the main goal in a late-stage study involving patients with Friedreich’s ataxia (FA), a rare, degenerative neuromuscular disorder that has no approved treatments.

The drug failed to demonstrate a statistically significant benefit over a placebo on an FA rating scale after 26 weeks, the Ireland-based company said.

Actimmune is already approved by the U.S. Food and Drug Administration for use in two rare, genetic conditions - chronic granulomatous disease and severe, malignant osteopetrosis.

Trading of the company’s shares were halted before the opening bell on Thursday. (Reporting by Natalie Grover in Bengaluru; Editing by Saumyadeb Chakrabarty)

Our Standards:The Thomson Reuters Trust Principles.
0 : 0
  • narrow-browser-and-phone
  • medium-browser-and-portrait-tablet
  • landscape-tablet
  • medium-wide-browser
  • wide-browser-and-larger
  • medium-browser-and-landscape-tablet
  • medium-wide-browser-and-larger
  • above-phone
  • portrait-tablet-and-above
  • above-portrait-tablet
  • landscape-tablet-and-above
  • landscape-tablet-and-medium-wide-browser
  • portrait-tablet-and-below
  • landscape-tablet-and-below