Breakthrough Patient Recruitment

: Chagas and Benznidazole: A Treatment that Started Out as a Donation

Chagas and Benznidazole: A Treatment that Started Out as a Donation

Online Media Specialist

One year ago, the Healthyist wrote about how Ebola could potentially bring much needed attention to the FDA's priority review voucher program (PVR) if it was added to WHO's list of neglected tropical diseases (NTD). As strange as it seems, the PVR law has come back into the limelight via another extremely serious disease, Chagas. Unlike Ebola, Chagas (American Trypanosomiasis) isalready classified by WHO as a NTD.

When a disease is categorized as a NTD, it generally affects a large portion of people in third-world countries but only a small percentage in more developed countries. Because of this, first-world countries with the technology to develop treatments have less incentive to invest millions on something from which they will not profit. In steps in the FDA's PVR program. My post from last year discusses how one of the two FDA PVR vouchers can be sold for millions by the receiving pharmaceutical company. This money is meant to recoup losses put into research and development of the treatment, which can easily reach more than 100 million. Although there has been some misinformation about how the PVR program works, one thing is for sure: it does not allow a company to skip clinical trials on the road to FDA approval. Rather, the PVR program is actually just an expedited process at the FDA review level. So, where does benznidazole fit in with this?

Chagas was only recently added to the NTD list of qualifying PVR diseases but already has two main treatments: benznidazole and nifurtimox. One of these treatments has recently started popping up unexpectedly in the news. This new interest in benznidazole has brought to light several underlying subplots that could have a positive effect on the pharma industry (weird, I know, stay with me.) First, it renews interest in a disease still affecting millions in poverty stricken countries. Search queries using the term Chagas have risen by more than 100 percent in December 2015 compared to prior months. Second, more attention is being brought to the PVR program and is already causing a federal systematic review of the important policy (which is still pretty new, as FDA programs go). And finally, buried deep within copy text, is the fact that this life-saving drug was originally an act of good will from another pharma company.

Neither benznidazole nor nifurtimox is currently approved by the FDA, but benznidazole is available for free via the CDC in the United States for qualifying patients. Benznidazole has not been approved by the FDA for sale in the United States yet due to the fact that the number of U.S. patients with Chagas who require treatment is relatively low (a reason why Chagas was added to NTD list). The leader of the CDC program that provides benznidazole reports that the CDC only provided 60 to 70 courses of treatment of either benznidazole or nifurtimox over the past year in the United States. But the disease is considered to be an epidemic in South America and causes approximately 11,000 deaths a year in Latin America.

That rate is steadily declining due to the fact benznidazole or nifurtimox is widely available in a number of Latin American countries--thanks to a donation. Benznidazole was originally developed by the pharmaceutical company Roche in the 1970s but was later donated by the company during the beginning of the Chagas outbreak in several South American countries. The donation transfer to the Brazilian government included the rights, trade data, and knowledge and technical information to manufacture its medicine.

After an agreement was made to subcontract manufacturing and marketing to a public Brazilian agency, the Laboratorio Farmaceutico do Estado de Pernambuco (LAFEPE), from 2003 to 2008, Roche worked to transfer the necessary technology to develop the drug to LAFEPE. In 2009, through a partnership with LAFEPE and DNDi, the first pediatric formulation of benznidazole became available in 21 countries in Central and South America. The partnership allowed the drug to be sold at cost, with no profit to the institutions involved, keeping the price of the two-month treatment at $50 to $100 USD.

Roche helped to save the Brazilian government and Latin American medical industry millions of dollars in research and development for a disease plaguing its low income regions. Production of generic forms of benznidazole, with the same active ingredient founded by Roche, has expanded to Argentina in order to meet an ever growing demand, while still keeping pricing down.


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