DNDi statement on KaloBios’ intention to raise price of Chagas drug and file for FDA priority review

[New York, December 14, 2015]
As reported this weekend in the New York Times, the pharmaceutical company KaloBios has announced its intention to register the drug benznidazole with the United States Food and Drug Administration (FDA) and to charge exorbitant prices for this treatment, which is an essential medicine for Chagas disease, the leading parasitic killer in Latin America. By registering this drug in the U.S., KaloBios also hopes to obtain a neglected disease “priority review voucher,” which it would likely be able to sell for a large sum – the latest “priority review voucher” was sold for $350 million.

Although details about this move are still emerging, documents filed with the US Securities and Exchange Commission (SEC) show that KaloBios intends to price benznidazole at levels “similar to Hepatitis C antivirals,” which are some of the most highly-priced drugs on the planet. Currently, benznidazole is available in many Latin American countries for $60-100, while the new generation of Hepatitis C antivirals cost up to $94,000.

At this point, we see the move by KaloBios as a direct threat to access to affordable benznidazole both in the U.S. and Latin America,” said Dr Bernard Pécoul, Executive Director of the Drugs for Neglected Diseases initiative (DNDi). “Sustainable access to Chagas treatment is an urgent public health priority that will be deeply compromised if benznidazole is priced out reach for those who need it most.”

An estimated 5.7 million people are infected with Chagas disease, which is transmitted by the bite of the triatomine, also known as the ‘kissing bug’, a blood-sucking insect found in the Americas. It can also be transmitted from mother to child or through blood transfusions, organ transplantation, and less frequently through foodborne exposure. Despite this burden, less than 1% of those in need currently receive treatment.

Benznidazole is one of only two drugs used to treat Chagas – the other is called nifurtimox and has serious side effects. Despite the need for improved treatments, benznidazole is currently a vital tool in the practically empty therapeutic toolbox to combat Chagas disease. The drug was developed in the 1970s (see timeline below) and is now on the Essential Medicines List of the World Health Organization (WHO).

Although the overwhelming majority of Chagas disease patients live in Latin America, DNDi and many others have long pushed for U.S. FDA approval of benznidazole to ensure broader access to treatment for the estimated 300,000 Chagas disease patients in the U.S. Unfortunately, the latest announcement about pricing by controversial biotech entrepreneur Martin Shkreli, who became the CEO of KaloBios after recently acquiring the company, does not bode well for these patients. The socio-demographics of Chagas patients in the US are poorly known. However, information suggests that many of them are likely to have little access to health services. Currently, benznidazole is available for free from the US Centers for Disease Control and Prevention (CDC) through a clinical trial protocol, but providers need to go through a lengthy and bureaucratic application process.

DNDi and others are equally troubled by KaloBios’ intention to apply for a FDA Priority Review Voucher (PRV) for benznidazole, a decades-old drug. Chagas was recently added to the list of eligible diseases for this program, which awards a voucher to companies that receive FDA approval for a drug to treat one of a list of neglected diseases. Vouchers can be used to fast track a drug application of the company’s choice or resold.

KaloBios has not conducted significant research and development (R&D) for benznidazole, nor does it intend to do so as per the company’s statements. The company appears to have the sole intention of registering benznidazole in the U.S. to obtain the lucrative PRV, without taking the necessary steps to ensure its affordability.

The FDA PRV program has the potential to be a powerful incentive for companies to conduct research and development for neglected diseases, but unfortunately there are serious loopholes that allow companies to get vouchers when they have barely invested into R&D, and to profit while not committing to make their products accessible to patients in need,” said Rachel Cohen, Regional Executive Director of DNDi North America. “We could face a nightmare situation for Chagas patients and health care providers in the US: the drug will finally be registered, but it could be even less accessible than it is today.”

DNDi, Médecins Sans Frontières/Doctors Without Borders (MSF), and other public health organizations recently detailed concerns about the PRV in an open letter to the U.S. Senate. One of the main demands was that the PRV program should require an access strategy to ensure patients, governments and health care providers have affordable and appropriate access to products for which a PRV has been awarded.

As we work to dramatically scale up access to diagnosis and treatment of Chagas disease with currently available treatments, R&D needs to be stepped up to develop new generation medicines and vaccines for Chagas that are safe, effective, affordable and accessible,” added Dr Pécoul.

Chagas disease is one of DNDi’s priority diseases and the organization is actively engaged in developing new treatments and screening new compounds. DNDi and partners delivered the first pediatric dosage form of benznidazole and is running clinical trials evaluating new regimens or combinations of the drug. Importantly, DNDi is deeply involved in early stage research for entirely new drugs for Chagas disease, including the Neglected Tropical Disease ‘Drug Discovery Booster’, which allows DNDi to simultaneously screen the compound libraries of four pharmaceutical companies.

 

Short Timeline of Benznidazole

1970s: Roche develops Benznidazole and registers it in several Latin American countries

2003: Roche decides to donate all commercial rights and technology to manufacture benznidazole to the Brazilian government, which produces a generic version of Roche’s product.

2006: Brazilian state laboratory LAFEPE gets marketing authorization for benznidazole

2008: DNDi and LAFEPE enter into a joint development program to develop a pediatric dosage form of benznidazole.

2011: DNDi and LAFEPE’s pediatric formulation, adopted for babies and children, is registered in Brazil.

2012: A consortium in Argentina develops 100 and 50mg dosages of benznidazole are produced and registered in several countries by the Argentinian company, Elea

2013: Pediatric benznidazole is included on WHO essential medicines list for children

2013: DNDi concludes the then first and only randomized placebo-controlled study using benznidazole in adults with Chagas disease, which provides key data and evidence about the efficacy of benznidazole.

2015: Results are published from another randomized-controlled study on the use of benznidazole for patients with chronic Chagas cardiomyopathy, a potentially fatal disease of the heart muscle caused by Chagas parasites. The study shows that benznidazole is effective in clearing the parasite from patients, but has no effect on slowing long-term heart disease. This confirms the need for early treatment of Chagas patients.

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Media Contacts:
Ilan Moss
imoss@dndi.org
+1 646 266 5216

Violaine Dällenbach
vdallenbach@dndi.org
+41 22 906 92 47