3 neglected-disease treatments newly added to WHO Essential Medicines List for paediatric use

(Press-News.org) [Geneva, Switzerland – 11 July 2013] – This week the World Health Organization (WHO) released its newly updated 4th WHO Model List of Essential Medicines for Children (EMLc), in which three treatments developed by the Drugs for Neglected Diseases initiative (DNDi) and its partners have now been included. One treatment was also added to the 18th WHO Model List of Essential Medicines (EML) for adults.

Artesunate-mefloquine fixed-dose combination (ASMQ FDC) was added to the EMLc for the treatment of malaria in children, and to the EML for adults, in line with current WHO treatment guidelines. ASMQ FDC was developed with Farmanguinhos/Fiocruz (Brazil) and launched first in Brazil in 2008. After a technology transfer to Cipla (India), ASMQ FDC was prequalified by WHO in 2012, and registered in India, Malaysia, and Myanmar in 2011-2013.

Nifurtimox-eflornithine combination therapy (NECT) was added to the EMLc for the treatment of late-stage sleeping sickness (human African trypanosomiasis) in children. Developed in partnership with Médecins Sans Frontières, Epicentre, and the Swiss Tropical and Public Health Institute, NECT was launched and added to the EML for adult treatment in 2009. NECT is the first new treatment option in over 25 years for sleeping sickness. It has been implemented and distributed by WHO - supported by donations by Sanofi and Bayer - through national control programmes in the 12 sub-Saharan African countries where 98% of late-stage sleeping sickness cases occur, replacing an old, toxic, arsenic-based drug that was commonly used before.

Paediatric dosage form of benznidazole was added to the EMLc for the treatment of Chagas disease (American trypanosomiasis) in children. This child-adapted formulation of benznidazole - the main drug used to treat Chagas disease - was developed in partnership with Lafepe (Brazil) as an easily dispersible tablet for simple and accurate oral use in young children. The treatment was registered in Brazil in 2011.

'Children are often the first victims of parasitic diseases in developing countries, so we are very pleased that three life-saving, neglected-disease treatments for paediatric use, developed by DNDi and our partners, have been added to the WHO's Essential Medicines Lists,' said Dr Bernard Pécoul, Executive Director of DNDi. 'This will facilitate access, quick adoption, and use by endemic countries to ensure they benefit young patients most in need.'

Updated every two years, the WHO's EML and EMLc serve as critical guides for informing country-level essential medicine lists, procurement and supply of medicines, and clinical decision-making.

The inclusions of these new paediatric therapeutic options to the WHO EMLc attest to the urgent, specific treatment needs of children threatened by neglected diseases such as African sleeping sickness and Chagas disease, and highlight the leading public-goods product-development work of DNDi in the area of paediatric research and development (R&D) for neglected diseases and patients.

### About WHO EML Main webpage: http://www.who.int/medicines/publications/essentialmedicines/en/ Executive Summary, July 2013: http://www.who.int/selection_medicines/committees/expert/19/EC19_Executive_summary_Final_web_8Jul2013.pdf

About ASMQ FDC The combination of artesunate (AS) and mefloquine (MQ), two well-established drugs for the treatment of uncomplicated P. falciparum malaria, has proven its efficacy after 20 years of clinical use. However, the non-fixed dose combination posed problems of patient compliance and potential development of drug resistance. In order to address this, ASMQ fixed-dose combination (FDC) was developed by the Fixed-Dose Artesunate-Based Combination Therapies (FACT) Consortium, created by DNDi and the WHO Special Programme for Research and Training in Tropical Diseases (TDR) in 2002. ASMQ FDC was developed through an innovative partnership with the Brazilian public pharmaceutical company Farmanguinhos/Fiocruz and was registered in Brazil in 2008, India in 2011, and Malaysia and Myanmar in 2012. ASMQ FDC is prequalified by WHO (meets standards of quality, safety, and efficacy). Through a technology transfer from Farmanguinhos to the Indian generic-drug manufacturer Cipla, ASMQ FDC is also produced and available in Asia. It is easy to use, with once daily administration of one or two tablets over three days for patients of all ages (from children aged 6 months through to adults) and has a two-year shelf-life in tropical conditions. http://www.dndi.org/treatments/asmq.html

About NECT Launched in 2009, nifurtimox-eflornithine combination therapy (NECT) was the first new treatment in over 25 years for late-stage human African trypanosomiasis (sleeping sickness). It consists of co-administration of oral nifurtimox tablets (10 days) and intravenous (IV) infusions of eflornithine (14 infusions over 7 days). NECT was added to the WHO Model List of Essential Medicines (EML) in 2009 and is on the essential medicines list of 12 African countries that account for 98% of reported cases of sleeping sickness. Over 60% of all late-stage sleeping sickness patients in endemic countries (2011), and 96% in the Democratic Republic of the Congo (DRC; 2012), the country with the most cases, were treated with NECT. NECT was the result of a six-year collaboration among DNDi, Médecins Sans Frontières/Doctors Without Borders (MSF), Epicentre, Swiss Tropical and Public Health Institute, and the sleeping sickness national control programmes of the DRC and Republic of Congo, with support from WHO. The drugs are donated by Sanofi and Bayer, and the treatment kits are prepared and distributed by MSF Logistique. http://www.dndi.org/treatments/nect-c-treatments.html

About Paediatric Dosage Form of Benznidazole The paediatric dosage form of benznidazole was launched in December 2011 for the treatment of Chagas disease in children. The age-adapted 12.5-mg dispersible tablet is easy to use, affordable, and non-patented, designed for use in infants and young children under 2 years of age (20 kg body weight). Treatment is designed to use one, two, or three tablets, depending on weight (recommended dosage, 5-10 mg/kg body weight/day). Since the paediatric tablet is easily disintegrated and requires no tablet fractionation (pill cutting), treatment of children is simplified, with improved dosing accuracy, safety, and adherence. The new treatment was the result of a collaboration between DNDi and LAFEPE (Pernambuco State Pharmaceutical Laboratory/Laboratório Farmacêutico do Estado de Pernambuco), the second largest public laboratory in Brazil. The paediatric dosage form was granted registration by Brazil's National Health Surveillance Agency (ANVISA), and available for procurement through the PAHO Strategic Fund and LAFEPE. Tools to facilitate implementation of and access to the new treatment include a Demand Forecasting Planning Tool, Procurement Guide, and Tool Box of training and educational materials for doctors and caregivers. http://www.dndi.org/treatments/paediatricbenz.html

About DNDi The Drugs for Neglected Diseases initiative (DNDi) is a not-for-profit research and development (R&D) organization working to deliver new treatments for the most neglected diseases, in particular sleeping sickness (human African trypanosomiasis), Chagas disease, leishmaniasis, filaria, and paediatric HIV/AIDS. Since its inception in 2003, DNDi has delivered six new treatments: two fixed-dose antimalarials (ASAQ and ASMQ), nifurtimox-eflornithine combination therapy (NECT) for late-stage sleeping sickness, sodium stibogluconate and paromomycin (SSG&PM) combination therapy for visceral leishmaniasis in Africa, a set of combination therapies for visceral leishmaniasis in Asia, and a paediatric dosage form of benznidazole for Chagas disease. DNDi was established by Médecins Sans Frontières/Doctors Without Borders (MSF), Indian Council of Medical Research, Kenya Medical Research Institute, Brazil's Oswaldo Cruz Foundation, Ministry of Health of Malaysia, and Institut Pasteur in France, with the UNICEF/UNDP/World Bank/WHO Special Programme for Research and Training in Tropical Diseases (TDR) as a permanent observer. http://www.dndi.org

Media contacts:

Violaine Dällenbach
Press & Communications Manager, DNDi
e-mail: vdallenbach@dndi.org / Tel: +41 22 906 92 47 / Mobile: +41 79 424 14 74

Oliver Yun
Communications Manager, DNDi North America
e-mail: oyun@dndi.org / Tel: +1-646-616-8681 / Mobile: +1-646-266-5216