Drugs for Neglected Diseases Initiative

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The logo of the Drugs for Neglected Diseases Initiative.

The Drugs for Neglected Diseases Initiative (DNDI) is a collaborative, patients’ needs-driven, non-profit drug research and development (R&D) organization that is developing new treatments for neglected diseases, notably leishmaniasis, sleeping sickness (human African trypanosomiasis, HAT), Chagas disease,[1] malaria, paediatric HIV,[2] and specific helminth infections. DNDi`s malaria activities were transferred to Medicines for Malaria Venture (MMV) in 2015.

Led by its Executive Director Bernard Pécoul,[3] DNDi’s headquarters are in Geneva, Switzerland, with offices in Kenya, India, Brazil, Malaysia, Japan, the Democratic Republic of Congo, and an affiliate in the USA.


Neglected diseases affect a large proportion of the world's population and bring with them an immense economic and health burden.[4] A significant portion of diseases, most prevalent in impoverished or developing countries, are in fact preventable and/or treatable.[5]

The current drug R&D process in the pharmaceutical industry is based on a market-driven approach.[6] Between 1975 and 1999, only 1.1% of new therapeutic products had been developed for neglected diseases.[7] Between 2000 and 2011, only 4% of new therapeutic products were indicated for use with neglected diseases.[7] There is a lack of modern, safe and effective medications to treat these diseases. This lack can be attributed to the under-investment by the majority of research-based pharmaceutical companies in developing new treatments as well as public policy failures to prioritize such diseases.[4] In recent years, the situation has begun to improve with the emergence of product development partnerships (PDPs). PDPs adopt a collaborative approach to drug discovery and development. Some large pharmaceutical companies have since established dedicated research sites for poverty-related diseases and are working closely with PDPs on drug development activities.[4]

In 2003, seven organizations from around the world joined forces to establish DNDi: the Oswaldo Cruz Foundation from Brazil, the Indian Council of Medical Research, the Kenya Medical Research Institute, the Ministry of Health of Malaysia and France’s Institut Pasteur, Médecins sans Frontières (MSF) and the Special Programme for Research and Training in Tropical Diseases (TDR), which acts as a permanent observer to the initiative.

Key Achievements[edit]

To date, DNDi has delivered six new treatments and built a large drug pipeline for neglected diseases with both improvements on existing drugs and entirely new chemical entities (NCEs).[8]

Treatments delivered to date:

ASAQ for Malaria, 2007

Launched in 2007, this antimalarial product is a fixed-dose combination of artesunate/amodiaquine (ASAQ). It is the result of a partnership between DNDi and Sanofi. Produced in Morocco, this product is cheap[9] (available at only $.05 for children, $1 for adults), administered in a simple regimen (1 or 2 tablets per day for 3 days), meets the latest WHO guidelines for malaria treatment in Africa and was granted "pre-qualified" status in 2008. It is included in the WHO Essential Medicines List and Essential Medicines List for Children.

End 2011, ASAQ was registered in 30 sub-Saharan countries, in India, and in Colombia, and more than 120 million treatments had been distributed.[10][11] In 2011, it was available in 12 African countries that account for 99% of reported HAT cases.[12][13] By the end of 2015, ASAQ was registered in 35 countries and territories, and more than 400 million treatments had been distributed. A technology transfer agreement has been signed with industrial partner Zenufa in Tanzania in order to provide an additional source of ASAQ. ASAQ was handed over to the MMV Access and Product Management Team in May 2015.

ASMQ for Malaria, 2008

The second antimalarial treatment developed by DNDi is a fixed-dose combination of artesunate and mefloquine launched in 2008. Developed by an international collaboration within the FACT Project Consortium. It has a simple and adapted regimen, a 3-year shelf life and a very high compliance rate. ASMQ is produced in Brazil by Farmanguinhos/Fiocruz and thanks to a South-South technology transfer, it is now also produced by Cipla. The latter was granted "pre-qualified" status by the WHO in 2012 and included in the WHO Essential Medicines List and Essential Medicines List for Children in 2013. By 2015 it was registered in Brazil, India, Malaysia, Myanmar, Tanzania, Vietnam, Niger, Burkina Faso, Thailand and Cambodia. By the end of 2015 more than one million treatments had been distributed. ASMQ was handed over to the MMV Access and Product Management Team in May 2015.

NECT for Sleeping Sickness, 2009

NECT, a combination therapy of nifurtimox and eflornithine, is the first new, improved treatment option in 25 years for stage 2 (advanced stage) human African trypanosomiasis (HAT) also known as sleeping sickness. It is the result of a six-year partnership between NGOs, governments, pharmaceutical companies, and the WHO. It was launched in 2009 and included in the WHO Essential Medicines List and WHO Essential Medicines List for Children in 2009 and 2013 respectively. It requires shorter hospitalization than previous treatment, and is much safer than previously widely used arsenic-based Melarsoprol that killed about 5% of patients.

SSG&PM for Visceral Leishmaniasis, 2010

SSG&PM,[14] a sodium stibogluconate plus paromomycin combination therapy, is a shorter-course, cost-efficient treatment option against Visceral Leishmaniasis (VL) in East Africa available since 2010. It is the result of a six-year partnership between DNDI, the Leishmaniasis East Africa Platform (LEAP), the National Control Programmes of Kenya, Sudan, Ethiopia, and Uganda, Médecins Sans Frontières (MSF) and the WHO.[15] It was recommended by the WHO Expert Committee on the Control of Leishmaniasis in 2010 as the first-line treatment in East Africa, and more than 10,000 patients have been treated. Sudan, Ethiopia, South Sudan and Somalia have released revised guidelines recommending SSG&PM as the first-line treatment for VL.

Visceral Leishmaniasis Treatments in Asia, 2011

Single dose AmBisome® & paromomycin/miltefosine/AmBisome® combinations were recommended by the WHO Expert Committee on the Control of Leishmaniasis (2010). These treatments are less toxic than previous mainstay treatments, useful in areas of antimonial resistance, are shorter course and their cost is comparable with previous treatments. In 2010, a study investigating the three possible 2-drug combinations of amphotericin B (AmBisome), miltefosine and paromomycin was completed in India. All three combination treatments were shown to be highly efficacious (> 97.5% cure rate). A WHO Expert committee recommended these treatments to be used preferentially to current established monotherapy treatments for VL in South Asia. DNDi is working with TDR and WHO to facilitate their introduction and support VL elimination strategies.[16] DNDi conducted more studies, including a pilot project in the Bihar State of India (2012-2015) that demonstrated the safety and effectiveness of combination therapies based on AmBisome®, miltefosine and paromomycin at the primary healthcare level, and single dose AmBisome® at the hospital level. Based on the study results, the Indian National Roadmap for Kala-Azar Elimination in August 2014 recommended use of single dose AmBisome® as a first option treatment for the treatment of VL patients, with paromycin and miltefosine as a second option at all levels; a policy also reflected in Bangladesh and Nepal. this removal of miltefosine monotherapy is an important policy change. This project has been a collaboration with the National (Vector Borne) Disease Control Programmes of India and Bangladesh, MSF, TDR, OneWorld Health/PATH, Bihar State Health Society and the Indian Council for Medical Research.

Paediatric Benznidazole for Chagas disease, 2011

This is the only paediatric dosage treatment for Chagas disease, launched in 2011 through a collaboration between DNDi and LAFEPE (Laboratório Farmacêutico do Estado de Pernambuco). In November 2013, the Mundo Sano Foundation and DNDi signed a collaboration agreement to deliver a second source of the treatment in partnership with ELEA (producers of Abarax®). The paediatric dosage form of benznidazole is designed for infants and young children under 2 years of age (20 kg body weight) infected congenitally. Thanks to its age-adapted, easy-to-use, affordable, and non-patented tablet, the new treatment contributes to improved dosing accuracy, safety, and adherence to treatment. The paediatric dosage form of benznidazole was granted registration by Brazil`s National Health Surveillance Agency (ANVISA) in 2011, and further endemic countries are targeted for obtaining registration. It was included on the WHO Essential Medicines List for Children in July 2013


In 2003, DNDi won the BBVA Foundation Frontiers of Knowledge Award in the Development Cooperation category[17] for developing and delivering new treatments for poverty-related diseases including Chagas disease, sleeping sickness, malaria and leishmaniasis.

DNDi received the Carlos Slim Health Award in 2013. Created in 2008 by the Carlos Slim Foundation, the aim of the award is to distinguish the people and institutions who are committed to improving the levels of health among the population of Latin America and the Caribbean.

In 2013, The Rockefeller Foundation asked the global community to nominate organizations and individuals who were making a difference for poor and vulnerable populations through innovation.[18] From those nominations - and the votes of individuals around the world - The Rockefeller Foundation selected three winners of the 2013 Next Century Innovators Award. DNDi was one of the awardees.[19]

On December 11, 2015, DNDi won the national FINEP Award for Innovation.[20] The award was in recognition of an innovative R&D model that has delivered a new antimalarial drug developed in Brazil.[21]

Regional Clinical Trial Platforms[edit]

DNDi works with partners in disease-endemic countries to strengthen existing clinical research capacity and build new capacity where necessary. DNDi helped in setting up and works with four regional disease-specific platforms in Africa and Latin America. The Leishmaniasis East Africa Platform (LEAP) on leishmaniasis. The HAT Platform on sleeping sickness, or human African trypanosomiasis. The Chagas Clinical Research Platform (CCRP). The RedeLeish Network on leishmaniasis in Latin America.

Their mission is to define patient needs, taking into consideration the local conditions, bring together key regional actors in the field of health, reinforce clinical capacities in endemic regions, address infrastructural requirements where necessary and provide on-site training.[22]

Long-term Objective[edit]

DNDi plans to develop 16-18 new treatments by 2023.[23]

See also[edit]


  1. ^ Nature Outlook Chagas Disease supplement. Nature Supplement, 2010 June, Vol. 465, No. 7301 suppl. ppS3-S22
  2. ^ Lallemant, Marc; Chang, Shing; Cohen, Rachel; Pécoul, Bernard (18 August 2011). "Pediatric HIV - A Neglected Disease ?". The New England Journal of Medicine 365: 581–583. doi:10.1056/NEJMp1107275. Retrieved 2016-05-02. 
  3. ^ The Lancet, "Bernard Pécoul: championing the cause of neglected diseases" - August 2010
  4. ^ a b c Burrows, Jeremy (5 March 2014). "The role of modern drug discovery in the fight against neglected and tropical diseases". MedChemComm. doi:10.1039/C4MD00011K. 
  5. ^ "The 10/90 Report on Health Research 2003-2004" (PDF). www.globalforumhealth.org. 2004. 
  6. ^ Ioset, Jean-Robert (September 2011). "Drugs for Neglected Diseases initiative model of drug development for neglected diseases: current status and future challenges". Future Medicinal Chemistry. doi:10.4155/fmc.11.102. 
  7. ^ a b Pedrique, Belen (24 October 2014). "The drug and vaccine landscape for neglected diseases (2000–11): a systematic assessment". The Lancet Global Health. doi:10.1016/S2214-109X(13)70078-0. 
  8. ^ "Portfolio". www.dndi.org. 
  9. ^ "Malaria Treatment". 
  10. ^ Innovative public-private partnerships to maximize the delivery of anti-malarial medicines: lessons learned from the ASAQ Winthrop experience by Bompart F, Kiechel J-R, Sebbag R, Pecoul B. Malaria Journal. May 2011, 10:143 doi:10.1186/1475-2875-10-143
  11. ^ The initial pharmaceutical development of an artesunate/amodiaquine oral formulation for the treatment of malaria: a public-private partnership by Lacaze C, Kauss T, Kiechel J-R, Caminiti A, Fawaz F, Terrassin L, Cuart S, Grislain L, Navaratnam V, Ghezzoul B, Gaudin K, White N.J, Olliaro P, Millet P. Malaria Journal. May 2011, 10:142 doi:10.1186/1475-2875-10-142
  12. ^ NECT Is Next: Implementing the New Drug Combination Therapy for Trypanosoma brucei gambiense Sleeping Sickness by Yun O, Priotto G, Tong J, Flevaud L, Chappuis F. PLoS NTD, 2010 May, 4(5):e720
  13. ^ Nifurtimox-eflornithine combination therapy for second-stage African Trypanosoma brucei gambiense trypanosomiasis: a multicentre, randomised, phase III, non-inferiority trial by Priotto G, Kasparian S, Mutombo W, Ngouama D, Ghorashian S, Arnold U, Ghabri S, Baudin E, Buard V, Kazadi-Kyanza S, Ilunga M, Mutangala W, Pohlig G, Schmid C, Karunakara U, Torreele E, Kande V. In Lancet. 2009; 374:56-64.
  14. ^ Musa, Ahmed (19 June 2012). "Sodium Stibogluconate (SSG) & Paromomycin Combination Compared to SSG for Visceral Leishmaniasis in East Africa: A Randomised Controlled Trial". PLOS. PMC 3378617. 
  15. ^ Visceral leishmaniasis treatment: What do we have, what do we need and how to deliver it? By Lucio H. Freitas-Junior, Eric Chatelain, Helena Andrade Kim, Jair L. Siqueira-Neto. International Journal for Parasitology: Drugs and Drug Resistance, Volume 2, December 2012
  16. ^ Comparison of short-course multidrug treatment with standard therapy for visceral leishmaniasis in India: an open-label, non-inferiority, randomised controlled trial by Sundar S, Sinha P.K, Rai M, Verma D.K, Nawin K, Alam S, Chakravarty J, Vaillant M, Verma N, Pandey K, Kumari P, Lal C.S, Arora R, Sharma B, Ellis S, Strub-Wourgaft N, Balasegaram M, Olliaro P, Das P, Modabber F. Lancet, 2011 January, doi:10.1016/S0140-6736(10)62050-8.
  17. ^ "DNDi win BBVA Award". 
  18. ^ "Carlos Slim Award". 
  19. ^ "Next Century Innovators Award". 
  20. ^ "DNDi receives FINEP Award". 
  21. ^ "Finep Award". 
  22. ^ "Strengthening Capacity". 
  23. ^ "DNDi Business Plan". 

External links and publications[edit]