Bayer Submits New Pediatric-Formulated Nifurtimox for FDA Approval to Treat Chagas Disease in Children

January 30, 2020 /Bio ValleyBIOON/ --Bayer(Bayer) recently announced that it has submitted to the U.S. Food and Drug Administration (FDA) submitted an application for a new formulation of nifurtimox, a scored and dispersible tablet for the treatment of pediatric patients (0–18 years) with Chagas disease (also known as American trypanosomiasis), to facilitate weight-based dosing in neonates, infants, and children.

This application is based on the results of the Phase III CHICO clinical study. This is the largest clinical treatment study ever conducted in the pediatric population with this disease, and the results confirmed the safety and efficacy of the new formulation of nifurtimox for treating pediatric patients with Chagas disease. CHICO is a prospective, randomized, double-blind, historically controlled Phase III study conducted between 2016 and 2018 at 25 research centers in Argentina, Bolivia, and Colombia. A total of 330 pediatric patients with acute or chronic Chagas disease were enrolled to evaluate the efficacy, safety, and pharmacokinetics of scoreable and dispersible nifurtimox tablets.

The results showed that the 60-day nifurtimox treatment regimen was superior to historical placebo controls in terms of serological response one year after the end of treatment, thereby meeting the study’s primary endpoint. In the overall study population, the 30-day nifurtimox treatment regimen yielded a lower serological response compared with the 60-day regimen. Weight-adjusted dosing of nifurtimox demonstrated a favorable safety profile.

Dr. Jaime Altcheh, Coordinating Investigator of the CHICO study and Head of the Department of Parasitic and Chagas Diseases at Ricardo Gutiérrez Children's Hospital in Buenos Aires, Argentina, stated, “In fact, the younger the patient, the greater the chance of recovery. With the new pediatric formulation, we can treat neonates and infants more accurately. The tablets of this new formulation can be crushed to allocate doses. It is also important that these tablets dissolve in water very quickly, which is particularly crucial because these very young children cannot swallow solid tablets but must undergo pharmacological treatment three times daily for 60 days. The new nifurtimox formulation represents a significant step forward toward the goal of treating all infected children. Early treatment after infection is vital for preventing the manifestation of disease in adulthood.”

The goals of controlling Chagas disease are to eliminate transmission, control vectors, treat girls and women of childbearing age (to prevent transplacental transmission), and screen blood prior to transfusion. Furthermore, the aim is to ensure that infected individuals have access to medical care at all stages of the disease.

Treatment of Chagas disease relies solely on two nitroheterocyclic compounds, nifurtimox and benznidazole. To date, nifurtimox has been available only as 120-mg tablets, which are difficult to administer, particularly in young children. Bayer has now developed a 30-mg tablet. Both tablet formulations are rapidly dispersible, can be easily split, and dissolve quickly in water to form a suspension for administration to patients who have difficulty swallowing tablets. The new formulation has been successfully used in the CHICO study, improving dosing accuracy, safety, and treatment adherence in children across all age groups.

Stefan Oelrich, Member of the Bayer AG Board of Management and President of the Pharmaceuticals Division at Bayer, stated: “The new formulation of nifurtimox will improve administration in neonates, infants, and children—the most vulnerable patient population affected by Chagas disease—and will make a significant contribution to controlling the disease. We will enhance access to nifurtimox by submitting new applications in countries with a high disease burden.”

Chagas disease is caused by *Trypanosoma cruzi* and is primarily transmitted by blood-sucking triatomine bugs. It was discovered by Dr. Chagas in 1908, hence the name Chagas disease. The disease is mainly endemic to Central and South America, so it is also known as American trypanosomiasis. *Trypanosoma cruzi* parasitizes the blood and various tissue cells of humans and other mammals. Its life cycle includes two forms: trypomastigotes and amastigotes. Trypomastigotes invade the human bloodstream through breaks in the skin. The disease can also be transmitted via breast milk, across the placenta, through blood transfusions, or by ingesting food contaminated with the feces of infectious triatomine bugs. It is estimated that there are 20 million infected individuals in Central and South America.

Most infections are asymptomatic. Symptomatic cases present only with mild flu-like symptoms that appear immediately after infection. Approximately two months later, Chagas disease enters a chronic phase that persists for many years, leading to severe organ damage and ultimately sudden cardiac death. Education, earlyDiagnosisTreatment and prevention are key to Chagas disease.

Currently, due to insufficient disease awareness and limited access to treatment, less than 1% of patients infected with Chagas disease receive therapy. Bayer is committed to expanding access to and use of nifurtimox by filing new registration applications and providing new formulations in countries with a high disease burden. (Bioon.com)

Original Source:Bayer application for nifurtimox to treat pediatric patients with Chagas disease submitted for approval in US