Orphan Drug Designation Just Granted for New SBS Treatment

The United States FDA just granted Orphan Drug Designation to Napo Pharmaceuticals for Mytesi (crofelemer) in the treatment of short bowel syndrome (SBS).
SBS is a rare disorder in which individuals have poor function of the small intestine.

This makes it difficult for them to maintain water and get enough nutrients from the food they eat, resulting in a series of potentially life-threatening complications, such as diarrhea and malnutrition and dehydration. To learn more about SBS, click here.

There are supportive treatments currently available for patients with SBS, but Mytesi is different because it contains the active pharmaceutical agent, Crofelemer.
According to the chair of Napo Pharmaceuticals’ Scientific Advisory board, Dr. Pravin Chaturvedi, Crofelemer is an anti-secretory agent that is first-in-class. It has a physiological mechanism to regulate the functioning of chloride ion channels in the body, which help manage fluid loss in SBS patients.
Thus, Mytesi works to block a particular chloride channel in the gastrointestinal system. These has the effect of decreasing sodium and water excretion and improving the consistency of stool.
It targets a different pathway in the body to help manage SBS than other current treatment options, and with the new Orphan Drug Designation, Napo Pharmaceuticals will have various development incentives and seven years of market exclusivity to keep advancing the drug.
Stay tuned in the coming months as the journey to bring Mytesi to market continues. Hopefully, it will not be long before SBS patients can start experiencing better relief.

In the meantime, if you want to read more about this new designation for Mytesi, click here!

To find out more about SBS, check out our partner Avery’s Angels.

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