Tropical Disease PRV Fix Didn’t Stop Novartis From Winning Another

Regulatory NewsRegulatory News
| 18 February 2019 | By Zachary Brennan 

Back in 2017, a new law was enacted, known as the FDA Reauthorization Act (FDARA), which contained not only the new user fee programs for pharmaceuticals, medical devices, generics and biosimilars, but also a slight tweak to the tropical disease priority review voucher (PRV) program.

The change was meant to ensure that companies winning PRVs (which means that they can sell such PRVs for potentially $100 million) actually performed some of the clinical work to bring the tropical disease drug to market in the US, and that the drugs “were not submitted as part of an application for marketing approval or licensure by a regulatory authority in India, Brazil, Thailand, or any country that is a member of the Pharmaceutical Inspection Convention or the Pharmaceutical Inspection Cooperation Scheme.”

Part of the reason these changes were made is that companies had abused the previous system. For example, Novartis won the first-awarded PRV in 2009 for an antimalarial drug that critics contend had been available in developing countries for years and was first registered in 1999.

Similarly, this week, Novartis was rewarded with another PRV for winning FDA approval for Egaten (triclabendazole), which is used to treat people with fascioliasis.

Doctors Without Borders (MSF), while criticizing the PRV program in general for not working “as it was designed for people desperately in need of new and affordable medicines for neglected diseases,” also notes that triclabendazole was registered in Egypt in 1998 and later in France by 2002. Additionally, the drug was placed on the WHO Essential Medicines List since at least 2002, MSF said.

So why didn’t the changes made in FDARA stop Novartis from winning a PRV?

FDA explained to Focus: “These new eligibility requirements apply to applications submitted after September 30, 2017, but did not apply to Novartis’s application.” And Novartis confirmed to Focus that the application was submitted before that date. "The changes made to the tropical disease PRV program in FDARA (Sec. 611) do not apply to our application."

But a look at the approval letter shows "the first portion" of the company's new drug application (NDA) was "dated September 21, 2017," just nine days before the FDARA changes were to take effect. The letter also notes upfront how the NDA was dated 14 June 2018.

However, the company ran multiple clinical investigations of triclabendazole and submitted data from a collection of more than 20 clinical studies, supporting the efficacy and safety profile, Novartis said.

And unlike the rare disease drug Firdapse (amifampridine), a previously unapproved drug that was provided free of charge and now will cost $375,000 after FDA approval, Novartis said last year it renewed an agreement with the WHO to extend the drug’s donation until 2022, expecting to reach 300,000 patients per year.

Rachel Sachs, associate professor of law at Washington University in St. Louis, told Focus that there’s still a difference in type between the products winning PRVs for neglected tropical disease and rare pediatric disease treatments. The ones for rare pediatric diseases can charge higher prices, but that’s not the case in tropical diseases.

“It may well be that these [FDARA] reforms will be effective,” she added.

Editor's note: Article updated to offer more clarity on PRVs in general.


© 2022 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy