NovaBiotics has announced that its Lynovex (NM001) cysteamine for the treatment of cystic fibrosis has been granted orphan drug designation by the FDA. NovaBiotics is developing both oral and dry powder inhalation formulations of the drug which, according to the company, breaks down mucus and prevents formation of biofilms in the lungs of CF patients.
A Phase 2a trial of the oral form, which is intended to treat exacerbations, is underway, and the company says that it plans to start clinical development of the DPI in the second half of 2015.
NovaBiotics CEO Deborah O’Neil commented, “The orphan drug designation for Lynovex in the US is a major regulatory milestone for NovaBiotics and highlights the unmet need for effective and safe treatments for CF patients. Dosing of the first patients in the Aberdeen Phase 2a clinical trial is also a significant clinical milestone in Lynovex’s clinical development.”
O’Neil added, “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. This is a candidate therapy with the potential to significantly improve the health and quality of life for those affected by CF. Importantly, and unlike other treatments that are specific to particular CF mutations, Lynovex is intended for use by the entire CF patient population, not just sub-groups.”
Read the NovaBiotics press release.
