European Committee Releases Positive Opinion on Cystic Fibrosis Drug for Orphan Medicinal Product

European Committee Releases Positive Opinion on Cystic Fibrosis Drug for Orphan Medicinal Product

Canada-based biotech Arch Biopartners announced the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has released a positive recommendation for the company’s treatment for infections linked to cystic fibrosis (CF).

The COMP’s positive opinion will be sent to the European Commission for final approval of the orphan designation for Arch Biotech’s product AB569. The approval is expected to be completed in the next few weeks.

Arch is now organizing AB569’s manufacturing and toxicology program, and is planning to submit an Investigational New Drug (IND) application to the U.S. FDA in 2016. The FDA recently granted AB569 Orphan Drug status for the treatment of antibiotic resistant Pseudomonas aeruginosa pulmonary infections in CF patients.

P. aeruginosa is a major cause of bacterial respiratory infections in patients with CF or chronic obstructive pulmonary disease (COPD), as well as a common cause of pneumonia. Since CF patients are more predisposed to lung infections – given an abnormal mucus production in the lungs and airways – P. aeruginosa infections are common, being present in up to 40 percent of patients between 6 and 10 years old. By the age of 17, the frequency of P. aeruginosa infections increases to 60 percent, and between 25 and 34 years old, the frequency reaches nearly 75 percent.

The mucoid form of P. aeruginosa is often found in patients suffering from CF, and is a very difficult infection to treat, mostly due to its high antibiotics resistance and phagocyte-mediated killing. As soon as patients develop this mucoid form of P. aeruginosa infection, their general lung function quickly declines. AB569 is an investigational potential treatment for both mucoid and non-mucoid pulmonary infections that are antibiotic-resistant.

“The positive opinion by the COMP in favor 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,” Dr. Daniel Muruve, Arch’s chief science officer, said in a press release.

CF affects almost one person per every 10,000 citizens of the European Union throughout all its 27 countries, which is well below the orphan drug limit.

This particular Orphan Medicinal Product designation recommendation includes the two AB569 active ingredients: sodium nitrite and ethylenediaminetetraacetic acid (EDTA).

AB569 was first discovered by Dr. Daniel Hassett at the University of Cincinnati College of Medicine as a treatment for antibiotic-resistant bacterial lung infections – a persistent problem in CF patients.

“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,” Hassett said.

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