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US orphan status for NovaBiotics' CF drug

Originally posted by Selina McKee on PharmaTimes.com

NovaBiotics’ novel, experimental treatment for Cystic Fibrosis has been awarded orphan drug status by the US Food and Drug Administration.

Courtesy of PharmaTimes

First-in-class Lynovex (NM001) is being developed both as a pill, for acute exacerbations, and in inhaled form, for chronic use and maintenance, and is intended for tandem use with existing CF treatments to intensify their antimicrobial effects.

The pill is currently being tested in a Phase IIa clinical trial with dosing due to start this week, while Phase IIb studies in the UK and Europe are expected next year. Clinical studies of the inhaled form are pencilled in for the the second half of 2015.

“If the encouraging data we have achieved so far continues to translate in clinical use, we believe Lynovex will offer a breakthrough in the treatment of CF,” said the Aberdeen, Scotland-based firm’s chief executive Deborah O’Neil, and highlighted its potential to “significantly improve the health and quality of life for those affected” by the disease.

She also noted that, setting it aside from other treatments specific to particular CF mutations, “Lynovex is intended for use by the entire CF patient population, not just sub-groups”.

CF is a chronic, life-limiting disease that affects the lungs, digestive tract and other organ systems. It is estimated that more than 70,000 around the globe are affected by the condition.