Drug development companies such as NovaBiotics that are devoted to designing and developing treatments for patients with difficult-to-treat, medically unmet diseases are often faced with the hurdle of recruiting enough patients to conduct the clinical trials necessary to approve new treatments. Recently, the hindrance has been somewhat lessened for one drug in development by NovaBiotics: the company’s 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.
“The orphan drug designation for Lynovex in the United States is a major regulatory milestone for NovaBiotics and highlights the unmet need for effective and safe treatments for cystic fibrosis patients,” said Dr. Deborah O’Neil, CEO of NovaBiotics, in a news release. “Dosing of the first patients in the Aberdeen Phase IIa clinical trial is also a significant clinical milestone in Lynovex’s clinical development.”
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, as reported previously by Cystic Fibrosis News Today. 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. O’Neil. “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.”
The reason for the non-specificity of Lynovex to any particular mutation subtype lies in its mechanism of action. It is believed Lynovex breaks down excessive mucus in the airways, killing bacteria that cause persisting and recurrent respiratory infections. This slows further damage to lung tissue and improves lung function.
By breaking down the mucosal barrier, Lynovex potentiates the antimicrobial actions of existing cystic fibrosis treatments. It will be intended for use as a treatment for acute exacerbations in its oral form and as a chronic therapy in dry powder, inhalable form.
Orphan drug status is designed for therapeutics intended to treat rare diseases that affect fewer than 200,000 Americans. Cystic fibrosis affects approximately 30,000 children and adults in the United States, and only 70,000 individuals worldwide. Thus, Lynovex meets the criteria, and it was already considered an orphan drug in Europe since the end of 2011.