Brazil's ANVISA grants registration for child-adapted formulation of benznidazole for Chagas disease

Today, at the occasion of the 4th DNDi Partners' Meeting in Rio de Janeiro, Brazil, Dr Carlos Gadelha, Secretary of Science, Technology and Strategic Products, Brazilian Ministry of Health, announced that Brazil's National Health Surveillance Agency (ANVISA) granted registration of a new paediatric dosage form of benznidazole, developed through a partnership between the Pernambuco State Pharmaceutical Laboratory (LAFEPE) of Brazil and the Drugs for Neglected Diseases initiative (DNDi). Registration of this child-adapted formulation of benznidazole will be published on 12 December 2011.

This new tablet means easier-to-administer and safer treatment of Chagas disease in infants and young children under the age of two, as they will receive accurate dosage.

Until today, benznidazole was available only as a 100-mg tablet for adults. Treatment for young children required cutting adult pills into tiny slivers - up to 12 pieces depending on the child's weight - and crushing and mixing them with water or juice, to be administered twice a day for 60 days. This difficult and inefficient method often results in improper dosing, risks of increased side-effects or ineffective treatment, and treatment stoppages.

Chagas disease infects an estimated 8 to 10 million people, mostly in Latin America, and kills some 12,000 people each year, making it the leading parasitic killer in the Americas, where it causes more deaths than malaria. The Chagas parasite is primarily transmitted via the bite of the blood-sucking triatome bug, widely known as the 'kissing bug', which is often found in poor housing conditions. In addition to blood transfusion, organ transplant, or ingesting infected food, the parasite is also transmitted during pregnancy from mother to child.

This new dosage form for children represents real progress for several reasons. Children are at especially high risk of infection, with a majority of them born from infected mothers. It is known that early treatment using benznidazole in the first year of life can eliminate the parasite in more than 90% of infected newborns. Thus, babies infected with Chagas disease will benefit the most from this new paediatric tablet.

'Thousands of mothers with Chagas disease-infected babies will welcome this as more than just a pill', says Manuel Guti-rrez, President of the International Federation of Chagas Patients. 'Their voices have finally been heard.'

The new 12.5-mg tablet is easily dispersible (disintegrated) and adapted for babies and children up to two 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 bodyweight/day).

'The presentation of this paediatric dosage form of benznidazole, as a final result of the collaborative project between DNDi and LAFEPE, opens possibilities for effective treatment [-] for thousands of children', says Dr Mirta Roses Periago, Director of the Pan American Health Organization (PAHO). 'From now on, hope of an early cure for infection of the parasite that causes Chagas disease is a wonderful reality.'

Tools to facilitate implementation of and access to the new treatment include a Demand Forecast, a Procurement Guide, and a Tool Box providing training and educational materials for doctors, other health professionals, mothers, and caregivers regarding appropriate use of the treatment.

In 2008, DNDi and LAFEPE entered a joint development agreement for this dosage form. The new tablet will be produced by LAFEPE, a public pharmaceutical manufacturer run by the State of Pernambuco in Brazil and the sole global producer of benznidazole.

'We will exert all efforts, regarding commercial aspects, towards rapid patient access of this drug', says Luciano Vasquez, President of LAFEPE. 'It will be offered at cost to all public health institutions, including the Ministry of Health of Brazil.' Indeed, the paediatric dosage form will also be made available at cost to non-governmental organizations and philanthropic institutions.

The new paediatric dosage form has been granted registration from Brazil's National Health Surveillance Agency (ANVISA), and DNDi is collaborating with LAFEPE to make the drug widely available, notably by working to register the drug in Argentina, Bolivia, Colombia, and Paraguay - priority countries where Chagas disease prevalence is high and treatment is urgently needed.

'At a time when efforts are being made to secure production of benznidazole, we must ensure that this new product is made available in all endemic areas and that treatment of children is rapidly expanded, in addition to the treatment of adults', says Dr Bernard P-coul, Executive Director of DNDi. 'With this new tablet, we can reach a turning point for the treatment of children infected with Chagas disease and we have to move rapidly to get the drug to them.'