FDA Approves Treatment for Neglected Tropical Disease

Officials with the FDA have approved Novartis’ triclabendazole (Egaten) for the treatment of fascioliasis in patients age 6 years and older.

According to Novartis, this product is the only FDA-approved drug for people with this disease, also known as liver fluke infestation. Fascioliasis is recognized by the FDA as a neglected tropical disease, triggering the award of a Priority Review Voucher based upon this approval.

The company expects the availability of triclabendazole in the United States to have an impact worldwide.

"Novartis has a long-standing commitment to addressing global health challenges and supporting disease elimination efforts, in diseases such as leprosy, malaria and fascioliasis," said Vas Narasimhan, CEO of Novartis, in a prepared statement. "Today's FDA approval of Egaten is another important milestone that we believe will help further expand access to this 1-day treatment, taking us a step closer toward disease elimination."

Fascioliasis is a tropical disease that infects 2.4 million people worldwide with an additional 180 million at risk of infection. No continent is free from fascioliasis, and human cases have been reported from more than 70 countries, according to Novartis.

The condition is caused by 2 species of parasitic flatworms that can infect humans following ingestion of larvae in contaminated water or food. Left untreated, fascioliasis can result in considerable pain and discomfort, leading to poor quality of life and loss of productivity.

The acute phase of the disease is manifested with fever, abdominal pain, nausea, diarrhea and eosinophilia. The disease progresses to a latent phase with less symptoms, and ultimately into a chronic or obstructive phase. In children, fascioliasis can be a serious infection with high fever, enlarged tender liver, and anemia.

Triclabendazole is the only medicine for fascioliasis recommended by the World Health Organization (WHO) and is on the WHO Model List of Essential Medicines. It is supplied by the global agency during epidemic outbreaks and for periodic use in endemic countries.

FDA approval of the Novartis triclabendazole product is expected to facilitate drug licensing and import to other countries.

"This FDA decision is welcome news for millions who suffer or are at risk of fascioliasis and removes a major hurdle in expanding treatment to countries where it is most needed," said Dr Mwelecela Malecela, Director of the Department of Control of Neglected Tropical Diseases at the WHO, in a prepared statement.

Novartis has been donating triclabendazole to the WHO since 2005, helping to treat approximately 2 million patients with fascioliasis in more than 30 countries. In 2018, the company renewed its agreement with the WHO to extend the drug donation until 2022, and is expected to reach 300 000 patients per year.

Reference

Novartis receives FDA approval for Egaten® for the treatment of fascioliasis, a neglected tropical disease [news release]. Basel, Switzerland; February 13, 2019: Novartis website. https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-egaten-treatment-fascioliasis-neglected-tropical-disease. Accessed February 2019.