Novoteris, LLC., a clinical stage medical device and pharmaceutical developer, has received orphan drug designation from the Office of Orphan Products Development of US Food and Drug Administration (FDA) for use of inhaled nitric oxide in treatment of Cystic Fibrosis.
The US Orphan Drug Act is intended to encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. In addition to providing a seven year term of market exclusivity upon final FDA approval, orphan drug designation also positions Novoteris to be able to leverage a wide range of financial and regulatory benefits, including government and institutional grants, and waiver of FDA user fees for the potential submission of a New Drug Application.
"The FDA's decision to grant nitric oxide orphan drug designation for this indication signifies an important step for Novoteris," stated Alex Stenzler, President of Novoteris. "The marketing exclusivity that orphan drug designation imparts adds significantly to the existing patent estate acquired by Novoteris." Chris Miller, PhD, chief science advisor, noted that, "The results from our pilot trial demonstrated more than a 15 per cent increase in lung function following two weeks of therapy and we anticipate that we will be able to demonstrate similar outcomes in our phase II trials." Following the presentation of the data from the pilot trial, both the US Cystic Fibrosis Foundation and the European Cystic Fibrosis Society sent letters of support for the Orphan Drug designation.
Cystic fibrosis (CF) is an autosomal recessive genetic disorder that affects most critically the lungs. Difficulty breathing is the most serious symptom and results from frequent lung infections that are most often treated with lifelong inhaled antibiotics.
Nitric oxide, an endogenous molecule, is the most important signaling molecule in the body and plays an important role in every organ system, including the innate defense system against microorganisms. Exogenous nitric oxide has been demonstrated to be an effective antimicrobial agent against a broad spectrum of microbes, including drug resistant strains. A lower concentration of nitric oxide is already on the market from other manufacturers for the treatment of pulmonary hypertension in newborns. "This new application brings years of work with nitric oxide gas for antimicrobial uses to the clinical forefront," added Dr Miller.