European Medicines Agency Grants Experimental Cystic Fibrosis Drug Orphan Drug Designation

According to a press release from Aridis Pharmaceuticals, the Company has received Orphan Drug designation from the European Medicines Agency for its experimental cystic fibrosis-related lung infection treatment AR-501.

The European Medicines Agency (EMA) is the regulatory body for pharmaceuticals available in the European Union market — filling a role analogous to that of the US Food and Drug Administration (FDA).

About Cystic Fibrosis

Cystic fibrosis (CF) is a genetically inherited condition characterized by the buildup of thick and viscous mucous in ducts throughout the body. In otherwise healthy individuals, this mucous is normally thin and slippery, acting as a kind of lubricant. However, in cystic fibrosis patients, this normally fluid lubricant instead plugs and obstructs small ducts, causing what can be significant organ dysfunction and considerable damage. The lungs and pancreas show particular susceptibility.

Cystic fibrosis is caused by genetic mutation to a gene called CFTR. Normally, this gene codes for the production of a protein called cystic fibrosis transmembrane conductance regulator (or CFTR), which helps chloride move to the surface of cells, attracting water. When CFTR has a mutation, dysfunctional CFTR protein is produced, which is much less effective at transporting chloride and therefore much less effective at attracting water to cells. Without the water attracted by CFTR, mucous produced by these cells takes on viscous qualities.

CF can lead to serious health problems, including chronic infection (particularly of the lungs) and sometimes even respiratory failure. Some cystic fibrosis patients develop digestive complications like diabetes or blockages of the bile ducts.

There is currently no cure for cystic fibrosis, and treatment is focused primarily on providing relief of symptoms and controlling potentially dangerous complications. Many with CF live shortened lives, so researchers searching for new treatments feel particularly under the cosh.

About AR-501 and the EMA Orphan Drug Designation

AR-501 is an inhaled mixture of gallium citrate that, in concentrations developed by Aridis, is believed to provide «broad-spectrum anti-infective activity» in the lungs of cystic fibrosis patients. Since frequent lung infection is a persistent and serious health concern for cystic fibrosis patients, managing it is a vital step forward in improving the well-being of these patients.

Although AR-501 has received Orphan Drug designation from the European Medicines Agency (similar to the orphan drug program operated by the United States’ FDA), it is still considered to be highly experimental — the program is currently in phase 1/2 testing, which is primarily concerned with establishing safety profiles and dosing guidelines. It could be months before trials evaluating AR-501’s effectiveness are run and it could be years before it reaches market (if it ever does).


Frequent lung infections can be fatal for cystic fibrosis patients. What do you think of the development of medications hoping to treat this serious complication? Share your thoughts with Patient Worthy!

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