NovaBiotics’ first-in-class drug Lynovex (NM001) received Orphan Drug Status from the Food and Drug Administration, allowing the requirement for the number of patients in a clinical trial to be lessened.
Orphan drug status is designed for therapeutics intended to treat rare diseases that affect fewer than 200,000 Americans, such as CF, which affects approximately 30,000 children and adults in the United States, and only 70,000 individuals worldwide.
Lynovex is in Phase IIa trials, and the announcement of orphan drug designation coincided with the dosing of the first patients in the study, which is co-sponsored by the Cystic Fibrosis Trust. Next year, Phase IIb trials will be conducted in the United Kingdom and Europe.
“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 cystic fibrosis,” stated Dr Deborah O’Neil, CEO of NovaBiotics.
“This is a candidate therapy with the potential to significantly improve the health and quality of life for those affected by cystic fibrosis. Importantly, and unlike other treatments that are specific to particular cystic fibrosis mutations, Lynovex is intended for use by the entire cystic fibrosis patient population, not just sub-groups.”