The EMA is currently reviewing the completeness of the application before initiating the official review process. It is expected that the Committee for Orphan Medicinal Products (COMP) will begin review of the application in approximately 30 days and will provide an opinion on the application that is forwarded to the European Commission (EC) by the end of May 2016.
The EC will then decide whether or not to approve the orphan drug designation for AB569.
An orphan drug designation in Europe provides incentives to a pharmaceutical company to develop a medicine for a rare disease. Such incentives include protocol assistance, which allows the drug company to get guidance from the EMA on the types of clinical studies needed to demonstrate the quality, benefits and risks of the new medicine.
Other incentives include access to a centralized authorization procedure and lower regulatory fees whereby a company can make a single new drug application to the EMA, resulting in a single drug approval decision from the EC, valid in all European Union Member States.
Orphan medicines approved by the EC further benefit from ten years of protection from market competition from similar drugs targeting the same indication.
In November 2015, the US FDA granted orphan drug designation for the combination of two active ingredients of AB569: sodium nitrite and ethylenediaminetetraacetic acid. AB569 is to be administered to patients as a nebulized (inhaled) solution. AB569 was invented at the University of Cincinnati in the lab of Dr. Daniel Hassett.
The Clinical Need for a New Treatment for P. aeruginosa Pulmonary Infections
P. aeruginosa is a significant cause of bacterial respiratory infections in patients who have cystic fibrosis (CF) or chronic obstructive pulmonary disease. It is also a common cause of pneumonia.
The mean prevalence of CF is approximately 0.74 cases per 10,000 people among 27 European Union countries, which is well below the defined limit for a rare or orphan disease. The mucoid form of P. aeruginosa, often found in CF patients, is a very challenging infection to treat due to its high resistance to both antibiotics and phagocyte-mediated killing.
Once patients present with the mucoid form of P. aeruginosa, their overall lung function precipitously declines, resulting in a poor prognosis.
Thus, there is an urgent clinical need for the development of novel effective treatments in this area. AB569 constitutes an innovative potential treatment for dealing with mucoid and nonmucoid P. aeruginosa pulmonary infections that are resistant to traditional antibiotics.
Cystic Fibrosis
CF is an autosomal recessive genetic disease that causes abnormalities of the CF transmembrane conductance regulator (CFTR) protein. CFTR is a critical regulator of sweat, digestive fluids, and mucus production.
CF patients are predisposed to lung infections due to abnormal mucus production in the lungs and airways. P. aeruginosa infects 40% of CF patients between the ages of 6 and 10 years of age. By the age of 17, the frequency of infection increases to 60% and reaches approximately 75% of all CF patients between the ages of 25 and 34.