The Food and Drug Administration (FDA) has granted Italian pharmaceutical company Edmond Pharma with the Orphan Drug Designation for their mucoactive drug Erdosteine, a therapy used for the treatment of bronchiectasis.
According to information available on the FDA’s website, designating an Orphan Product grants special status to a drug to treat a rare disease or condition that affects fewer than 200,000 patients in the U.S., upon request of a sponsor. Orphan Designation qualifies the sponsor of the drug for various development incentives, including tax credits for qualified clinical testing.
Currently marketed in more than 40 countries for the treatment of various respiratory diseases, Erdosteine is the newest molecule in the class of mucoactive drugs, and it has already treated over 3,000 patients in 60 clinical trials. Now, the therapy will be used for the treatment of bronchiectasis, a rare disease, in the U.S., where there are currently no approved drug therapies for the disease available.
The American Lung Association describes bronchiectasis as a condition in which the lungs’ airways are abnormally stretched and widened, due to mucus blockage. Mucus increasingly builds up in the airways, allowing bacteria to grow and finally leading to infection.
Roberto Teruzzi, CEO of Edmond Pharma, highlighted his company’s partnership with New Jersey-based Alitair Pharmaceuticals, a partnership that allowed Edmond to achieve the orphan drug status. “The market potential of this new therapy in the United States is considerable and corresponds to a therapeutic unmet need. Indeed there are no other drugs approved by the FDA for the treatment of bronchiectasis, a condition which has a heavy impact on the quality of life of the people affected,” he said in a press release.
Erdosteine will now be used in studies targeting bronchiectasis in the United States as part of the clinical development plan aimed to obtain the final authorization of the product as orphan drug.