Press release |

Patient Access to Miltefosine in Developing Countries Not Secure

Patient Access to Miltefosine in Developing Countries Not Secure Despite Award of US FDA Priority Review Voucher Sold for USD 125 Million

DNDi and MSF urge Knight Therapeutics, Paladin, and Endo to commit to key drug access requirements for treatment of leishmaniasis worldwide

Geneva, Switzerland & New York, USA, 25 November 2014 —  Knight Therapeutics has landed a USD 125 million windfall after selling its Priority Review Voucher (PRV) granted for the registration of miltefosine (Impavido®), even while access to the life-saving drug remains unsecured. The Drugs for Neglected Diseases initiative (DNDi) and Médécins Sans Frontières Access Campaign (MSF) are urging the drug’s license-holders and manufacturers – Knight Therapeutics, Paladin, and Endo – to ensure broad, sustainable access to the drug for the millions of people who need it.

In March 2014, Knight Therapeutics was awarded a Priority Review Voucher by the US Food and Drug Administration (FDA) for registering miltefosine in the United States. The FDA PRV is an incentive that aims at rewarding research and development (R&D) for new treatments for neglected tropical diseases. However, the R&D for miltefosine use in treating leishmaniasis was largely conducted in the mid-1990s by the WHO/TDR (Special Programme for Research and Training in Tropical Diseases) and partners, with private and public funding. Since then, DNDi and partners have invested in clinical studies testing the drug. Neither Paladin/Endo, nor Knight Therapeutics, has invested significantly in the R&D for the drug, but Knight Therapeutics will now benefit to the tune of USD 125 million after selling its Priority Review Voucher to Gilead.

Miltefosine is a vital drug in the currently fragile treatment arsenal for both visceral and cutaneous forms of leishmaniasis, a disease with over 1.3 million new cases and up to 40,000 deaths each year from the visceral form. There are currently only four drugs included in the World Health Organization’s Essential Medicines List for the treatment of leishmaniasis globally, including miltefosine.

Knight Therapeutics holds the exclusive license to distribute miltefosine (Impavido®) for leishmaniasis in the USA, whereas Paladin/Endo have maintained exclusive distribution and manufacturing rights for the rest of the world. Access to miltefosine in low- and middle-income countries with the highest disease burden has been inconsistent over recent years, with drug shortages, large minimum purchase requirements by the manufacturers, and a lack of response to public tenders where the drug was much needed, including in endemic countries such as India. In addition to its current use in Asia, the drug is being tested by DNDi and partners in Latin America and in Africa.

DNDi and MSF urge Knight Therapeutics, Paladin, and Endo to respond to the health needs of patients suffering from this neglected disease and to commit immediately to the following:

  • Disclose the actual cost of production of the drug;
  • Price the drug at-cost or with a minimal profit margin to ensure sustainable production of the drug (regardless of ordered quantities);
  • Maintain the registration of the drug in all disease-endemic countries for both visceral leishmaniasis and cutaneous leishmaniasis; and
  • Support additional clinical studies to optimize the use of miltefosine, including pharmacovigilance, dosing in children, use in cutaneous leishmaniasis and other complicated dermal forms of the disease, as well as co-infection with HIV.

The blatant hindrances to patient access to miltefosine for this neglected disease should be examined to the same extent as, and in conjunction with, the important economic benefit that Knight Therapeutics has received for selling the PRV for R&D that the company did not carry out. The PRV mechanism, which aims to stimulate or at least reward drug development for neglected diseases, currently contains no access provisions and fails to ensure that only entities that invested in R&D are awarded the voucher. MSF, DNDi, and others outlined their concerns about the US FDA PRV mechanism in a letter to the US Congress in July this year and have called for improvements to its design and implementation. With the PRV mechanism currently being amended by Congress to extend its applicability to Ebola, the opportunity to improve this important incentive mechanism should not be missed.

For more information on the FDA PRV for milestone: BMJ 2014;349:g4665 - US incentive scheme for neglected diseases: a good idea gone wrong?

Letter sent to US Congress available upon request.