European Medicines Panel Favors ‘Orphan Product’ Designation for Drug to Treat CF Lung Infections

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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EMA panel decision

The European Medicines Authority’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending the designation of orphan medicinal product to Arch Biopartners Inc.‘s AB569 as a potential treatment of infections in cystic fibrosis (CF) patients.

AB569 was invented at the University of Cincinnati (UC) in the laboratory of Dr. Daniel Hassett. The drug combines two active ingredients, sodium nitrite and ethylenediaminetetraacetic acid (EDTA), both approved as safe for use in people. In tests, AB569 has shown a dramatic effect at killing the bacteria Pseudomonas aeruginosa both in vitro and in vivo, including antibiotic-resistant strains.

“This very positive development will serve to accelerate our transition into CF clinical trials that are already planned at the University of Cincinnati and Cincinnati Children’s Hospital,” said Dr. Hassett in a company news release. Arch Biopartners recently entered an exclusive license agreement with UC for commercial rights to the drug candidate.

P. aeruginosa is the most common pathogen in CF patients’ lungs, which are especially vulnerable to chronic infections. Its prevalence in infections is estimated to be 40 percent in CF patients between the ages of 6 and 10, a frequency that increases to 60 percent by age 17, and reaches about 75 percent between the ages of 25 and 34. Antibiotic resistance to these infections complicates treatment, often leading to a progressive decline in lung function.

COMP’s recommendation will be forward to the European Commission (EC) for final approval, which is expected shortly.

The U.S. Food and Drug Administration (FDA) has also designated AB569 an orphan drug for the treatment of antibiotic resistant P. aeruginosa pulmonary infections in patients with CF.

“The positive opinion by the COMP in favour of orphan medicinal status for the treatment of CF is indicative of the clinical need to combat antibiotic resistant airway infections often found in CF patients,” said Dr. Daniel Muruve, chief science officer of Arch Biopartners.

The company recently developed a manufacturing and toxicology program for AB569, and future plans include the submission of an Investigational New Drug Application to the FDA in the second half of 2016.