New Drug for Short Bowel Syndrome Gets Orphan Designation

The FDA this week granted orphan drug designation to glepaglutide for the treatment of short bowel syndrome (SBS), developed by Zealand Pharma A/S.

SBS is a rare disorder where an individual cannot get enough nutrients and water from the food they eat due to poor function of the small intestine. Symptoms include debilitating diarrhea, malnutrition, dehydration, fluid imbalances, and more.

Last August, the FDA also granted orphan drug designation to Napo Pharmaceuticals for Mytesi (crofelemer) in the treatment of SBS.

Earlier this year, Zealand Pharma announced its phase 2 clinical trial for glepaglutide met its target, reducing fecal wet weight output and has sustained proper efficacy.

Orphan drug designation is an important benchmark, because it means that the designated drug qualifies for certain benefits from the federal government, such as reduced taxes.

According to Britt Meelby Jensen, President and CEO of Zealand:

“The orphan drug designation for glepaglutide enables us to have a closer and more frequent dialogue with the FDA throughout our late stage clinical development. Glepaglutide is one of Zealand’s leading product candidates, which we are developing all the way to market, and this designation is an important step for us in our efforts to ensure an efficient path to registration and patient access,”
Glepaglutide is set to begin phase 3 clinical trials in 2018.

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