According to a publication from Scleroderma News, the American Food and Drug Administration (FDA) recently granted Breakthrough Therapy status to Ofev (generic name nintedanib), as a treatment for progressive fibrosing interstitial lung disease.
Breakthrough Therapy designation is awarded to certain experimental treatments that, during clinical trials, show promise in significantly improving upon existing treatment methods. Although there is no guarantee that drugs assigned Breakthrough Therapy designation will gain market approval, the special status does speed along research and development. In theory, Breakthrough Therapy designation can speed up the drug development process even if the FDA ultimately rejects a candidate drug.
About Progressive Fibrosing Interstitial Lung Disease
Fibrosing interstitial lung disease (ILD) is actually a general term for a wide and diverse array of lung disorders that have a variety of causes, some of which remain unidentified. Although these disorders may originate in different conditions, there is often a significant degree of overlap in symptoms. Ultimately, however, all ILDs are similar in that they frequently result in functional lung decline, loss of quality of life, and often early mortality.
Progressive fibrosing interstitial lung disease is a term used to describe certain cases of ILD that are particularly severe and are characterized by persistent disease progression — sometimes even through treatment. In these cases, the scarring of the lungs can become self-sustaining, effectively creating a vicious cycle of fibrosis that can be exceptionally difficult to treat.
Certain common types of progressive ILD include idiopathic pulmonary fibrosis, chronic sarcoidosis, and nonspecific interstitial pneumonia. Sometimes, environmental factors like particulate exposure can contribute to the development of certain types of ILD — asbestosis and silicosis, for example.
For most progressive fibrotic ILDs, the standard of care is immunosuppression. However, once lung fibrosis becomes self-sustaining, immunosuppression is no longer effective — the need for more targeted drug therapies is clear.
About Ofev and Breakthrough Therapy Designation
Ofev (and nintedanib) is an orally-administered small molecule drug that inhibits the activity of tyrosine kinases — enzymes that are responsible for activating and deactivating numerous important cellular functions. Ofev has already been approved for idiopathic pulmonary fibrosis, where it is used to block the activity of a group of growth factor receptors that drive scarring.
Last month, Ofev received an additional FDA approval — this time for the treatment of systemic sclerosis-associated interstitial lung disease. It seems that Boehringer Ingelheim is endeavoring to diversify Ofev for use in as many ILDs as possible.
The FDA Breakthrough Therapy designation was granted following positive results from a phase 3 study of Ofev in a group of progressive ILD patients without consistent primary diagnoses. Drugs that receive Breakthrough Therapy designation are eligible for benefits provided by Fast Track designation. Research programs for breakthrough therapies are also entitled to organizational and management assistance from the FDA.
In studied progressive ILD patients, those who received Ofev demonstrated improved lung function retention after a period of one year. On average, those who received Ofev suffered 57% less loss of forced vital capacity, a standard metric for assessing lung health.
No targeted drug therapies are available for the majority of ILD subtypes. What do you think of this exciting announcement? Share your thoughts with Patient Worthy!