Bayer AG’s Lampit (nifurtimox) to treat Chagas disease in pediatric patients, approved by the FDA on Friday, is an oral, antiprotozoal medication for newborns to patients under 18, who weigh at least 2.5 kg and are diagnosed with Chagas disease caused by Trypanosoma cruzi.

Lampit is delivered by a dividable tablet that can be split by hand. The medication is formulated to disperse in water for patients with difficulty swallowing whole or halved tablets.

According to the World Health Organization (WHO), there are between 6 million and 7 million people infected with Chagas disease (American trypanosomiasis) globally. The illness can be acute or chronic. Symptoms include fever, fatigue, body aches, swollen glands and enlargement of the liver or spleen. More than 10,000 deaths per year are attributed to the disease.

“Chagas disease is mainly endemic in areas of 21 continental Latin American countries, where it is mostly … transmitted to humans by contact with feces or via the urine of triatomine bugs, known as ‘kissing bugs,’ among many other popular names, depending on the geographical area,” the organization said.

Lampit’s approval is based on a prospective, randomized (to dosing regimen), double-blind phase III study of the efficacy, safety and pharmacokinetics of nifurtimox in 330 male and female children with Chagas disease at 25 sites in Argentina, Bolivia and Colombia between 2016 and 2018. Bayer billed the study as the largest clinical trial ever in pediatric Chagas disease patients.

“In fact, the younger the patient, the better the chances of recovery,” said Jaime Altcheh, head of the Department of Parasitology and Chagas disease at the Ricardo Gutierrez Children’s Hospital in Buenos Aires and the clinical trial’s coordinating investigator.

The primary outcome measures were the percentage of cured patients at 12 months post-treatment and the incidence rate of seronegative conversion for subjects receiving at least one dose of the 60-day nifurtimox regimen. More than 300 patients with serologic evidence of T. cruzi infection, without Chagas disease-related cardiovascular and/or gastrointestinal symptoms, were randomly assigned to receive either a 60-day (n=219) or a 30-day (n=111) treatment regimen.

The study, CHICO (CHagas disease In Children treated with NifurtimOx), the first part of a larger phase III, met its primary endpoint, the serological response at one year after the end of treatment, by demonstrating superiority of the 60-day nifurtimox treatment compared with historical placebo control. Safety and efficacy were evaluated throughout the clinical trial.

The study will continue with a second part that includes patient follow-up.

Leverkusen, Germany-based Bayer said commercially insured patients may qualify for a $0 copay and uninsured patients who cannot afford the treatment may be eligible to receive it free through the Bayer U.S. Patient Assistance Foundation.

The WHO’s 21st List of Essential Medicines lists nifurtimox in 30-mg, 120-mg and 250-mg tablets for treating Chagas disease. The 120-mg dose of nifurtimox is also on the list for treating first-stage African trypanosomiasis. The dosage is only to be used in combination with eflornithine for treating Trypanosoma brucei gambiense infection. Medicines determined to be minimum needs for basic health care systems are selected for the list.

Nifurtimox is registered in Argentina, Uruguay, Chile, El Salvador, Guatemala and Honduras for treating acute and chronic Chagas disease in adults and children.

There are others developing Chagas disease treatments, including San Diego’s Selva Therapeutics Inc., which raised $3 million in a series A financing in late July. The company is driving SLV-213, a broad-spectrum antiviral for infectious diseases that include Chagas but also Ebola and Nipa viruses.

In April, Daiichi Sankyo Co. Ltd. renewed a joint research agreement with the Drugs for Neglected Diseases initiative for a new research program, the Lead Optimization Project, to develop treatments for Chagas disease. That project is funded through the Global Health Innovative Technology Fund.

7 Hills Pharma Inc., of Houston, is developing a small-molecule integrin activator, 7HP-349, that has shown preclinical vaccine responses to Chagas disease and tuberculosis.

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