FDA grants orphan status to Mytesi for short bowel syndrome

The FDA has issued orphan drug designation to Mytesi for the treatment of infants and children with short bowel syndrome, the manufacturer announced.

Mytesi (crofelemer, Napo Pharmaceuticals, Jaguar Health) is FDA-approved for treating the symptoms of noninfectious diarrhea in adults with HIV/AIDS on antiretroviral therapy.

Orphan status provides development incentives and 7 years of marketing exclusivity to the sponsor of an approved drug that treats a rare condition.

A press release from the drug manufacturer describes short bowel syndrome as “a complex condition characterized by malabsorption of fluids and nutrients due to congenital deficiencies, association with premature birth, or surgical resection of small bowel segments, [causing] debilitating diarrhea, malnutrition, dehydration and imbalances of fluids and salts.” It is more common in certain countries including the United Arab Emirates and Saudi Arabia, the release noted.

“Crofelemer, the active pharmaceutical ingredient in Mytesi, is a first-in-class anti-secretory agent with a novel physiological mechanism of action of regulating the functions of the chloride ion channels — which we believe may have considerable potential for managing the fluid loss and dehydration associated with chronic diarrhea that leads to devastating health implications in these pediatric patients,” Pravin Chaturvedi, PhD, chair of Napo’s scientific advisory board, said in the press release.

“With the early and extreme morbidity and mortality suffered by [short bowel syndrome] patients, we welcome the opportunity to participate in the investigation of a novel drug to address the devastating diarrhea and dehydration caused by this lifelong disease for which there is currently no available treatment except parenteral nutrition, and help limit the suffering of patients and their family members,” Mohamad Miqdady, MD, chief of pediatric gastroenterology, hepatology and nutrition at Sheikh Khalifa Medical City in Abu Dhabi, and a medical advisor to Napo, said in the press release.

Jaguar, which holds global rights to Mytesi’s key indications, estimates the potential U.S. market for the drug’s currently approved indication to be $100 million in gross annual sales and $7 million in revenue by April 2018. The company is pursuing a follow-on indication of chemotherapy-induced diarrhea. In addition to the orphan drug indication for short bowel syndrome, the drug is being developed as a second-generation anti-secretory agent for use in cholera patients, and for supportive care in patients with irritable bowel syndrome and inflammatory bowel disease.

Disclosures: Chaturvedi and Miqdady are employed by Napo.