[Ip-health] Patient access to miltefosine in developing countries not secure despite award of US FDA Priority Review Voucher sold for US$125 Million

Joanna Keenan-Siciliano joanna.l.keenan at gmail.com
Tue Nov 25 07:29:09 PST 2014

Patient access to miltefosine in developing countries not secure despite
award of US FDA Priority Review Voucher sold for US$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 US$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 $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

- ends -

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

Joanna Keenan
Press Officer
Médecins Sans Frontières - Access Campaign
P: +41 22 849 87 45
M: +41 79 203 13 02
E: joanna.keenan[at]geneva.msf.org
T: @joanna_keenan


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