Breakthrough Therapy designation for potential new anti-relapse medicine for malaria

US FDA grants expedited approval status for tafenoquine

20 Dec 2013

GlaxoSmithKline (GSK) and Medicines for Malaria Venture (MMV) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation* for tafenoquine, an investigational medicine for the treatment and relapse prevention of Plasmodium vivax malaria. Tafenoquine is not yet approved or licensed for use anywhere in the world. Breakthrough Therapy designation is the newest of the FDA’s programmes aimed at accelerating the development and review times of drugs for serious or life-threatening conditions.

P. vivax malaria is a neglected tropical disease and a major cause of uncomplicated malaria. It has a significant public health and economic impact primarily in South and South East Asia, Latin America and the horn of Africa, where the majority of the estimated 70-390 million annual clinical cases occur. The P. vivax parasite causes relapses from a dormant liver form established immediately after an infected mosquito bite. This dormant form leads to the reappearance of clinical malaria anywhere between a few weeks and several months after the initial infection. There is a need to provide alternative treatments to manage P. vivax relapse with shorter treatment regimens.

The Breakthrough Therapy designation was granted based on the results from an international, multicentre, randomised Phase II clinical trial in more than 300 patients with uncomplicated P. vivax malaria. Headline results from this trial were presented at the American Society of Tropical Medicine and Hygiene Meeting in November 2013, and detailed results published in The Lancet in December 2013. Plans are underway to start a Phase III study in 2014.


* The Breakthrough Therapy designation was enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA). The goal is to expedite the development and regulatory review of designated drugs to treat serious or life-threatening medical conditions when the drug may have substantial improvement over available therapies. Breakthrough Therapy designation includes all the features of the Fast Track designation, as well as more intensive guidance from the FDA on a drug’s clinical development programme.