A Potential Treatment for Idiopathic Pulmonary Fibrosis Earns Orphan Drug Designation

According to a story from Financial Buzz, the biotherapeutics company Bellerophon Therapeutics Inc. has recently announced that it has earned Orphan Drug designation from the US Food and Drug Administration (FDA) for nitric oxide as a treatment for idiopathic pulmonary fibrosis, a rare scarring disease that causes progressive decline in lung function. The company is currently testing its INOpulse system and nitric oxide against idiopathic pulmonary fibrosis in phase 2/3 clinical trial.

About Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic pulmonary fibrosis is a deadly, chronic, progressive lung disease which is characterized by lung tissue scarring, leading to a decline in lung function over time. The cause of the illness is unknown. With that being said, there are a few risk factors that have been identified, such as smoking cigarettes, exposure to various dusts (metal, wood, stone, and coal dust), occupations related to farming, family history, and potentially certain viral infections. Symptoms include shortness of breath, a dry cough, a distinctive crackling sound detected with a stethoscope, oxygen deficiency in the blood, and clubbed digits. There are few treatment options that can have a significant impact on the progression of idiopathic pulmonary fibrosis. Treatment may include certain medications, pulmonary rehabilitation, oxygen therapy, and lung transplant. Early intervention can make a major difference in outcomes; five year survival rate is between 20 and 40 percent. To learn more about idiopathic pulmonary fibrosis, click here.

About Orphan Drug Designation

Orphan Drug designation is typically reserved for therapies that are in development for the treatment of conditions and diseases that are considered rare. This is defined as any illness that affects less than 200,000 patients in the US. To qualify, a drug must display potential advantages in safety or efficacy in comparison to current treatments or must satisfy a medical need that is not being met by any available therapy. Recipients of Orphan Drug designation receive several benefits, such as tax breaks, the waiving of certain fees, and a period of market exclusivity lasting seven years if the drug earns market approval from the FDA.

Hopefully this treatment will be able to make a difference in the lives of these rare patients.


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