The European Commission, acting on the recommendation from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA), granted orphan medicinal product designation to AIT-CF, Advanced Inhalation Therapies’ (AIT) proprietary high dose formulation of nitric oxide (NO), for the treatment of cystic fibrosis. The Orphan Designation is held by AIT's European agent, Biological Consulting Europe Ltd, which is located in the United Kingdom.
AIT has completed a phase IIa trial in adult CF patients. In February 2015, the Company disclosed that the US Food and Drug Administration (FDA) also granted Orphan Drug Designation to AIT-CF for adjunctive treatment of cystic fibrosis (CF).
Dr David Greenberg, chief medical officer of Advanced Inhalation Therapies, commented, "AIT-CF potentially affords a significant benefit to CF patients. As a naturally occurring chemical in the body, NO, when inhaled at high concentrations, kills bacteria and fungi in the lungs of CF patients and has shown broad anti-infective activity. The European Orphan Drug Designation for AIT-CF affords additional benefits as we continue to advance its clinical development."
The European Commission's Orphan Designation is a status assigned to a medicine intended for use against a rare condition (prevalence of the condition in the European Union (EU) must not be more than 5 in 10,000). At the time of the designation, cystic fibrosis affected approximately 0.8 in 10,000 people in the EU or the equivalent of around 41,000 people based on current estimates. The EMA Orphan Medicinal Product Designation allows a pharmaceutical company to benefit from incentives offered by the EU to develop a medicine for the treatment, prevention or diagnosis of a disease that is life-threatening or a chronically debilitating rare disease. In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Medicinal Product Designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and gives direct access to centralized marketing authorization route.
Cystic Fibrosis (CF) is a lifelong, hereditary disease that causes mucus to form in the lungs and other organs. In 90% of CF cases, this mucus blocks the airways in the lungs, making it hard to breathe and leading to serious lung infections. For hospitalized CF patients, current treatments are supportive through the administration of oxygen through inhalation with bronchodilators, and include broad spectrum antibiotics and sometimes systemic steroids. Because these treatments do not eliminate all microbes, patients often suffer recurrent infections causing lung damage and ultimately in many cases respiratory failure.
AIT is a biopharmaceutical company developing novel anti-viral therapeutics based on nitric oxide. The company is applying its therapeutic expertise in NO to address significant medical needs, many of which cannot effectively be addressed with current standards of care, including small molecules and antibodies.