Neglected Tropical Diseases

Why are Neglected tropical diseases a priority for Novartis?

Neglected tropical diseases (NTDs) are a group of conditions that have a devastating impact on communities in low- and middle-income countries and affect more than one billion people worldwide.¹

Yet, NTDs only make up a small fraction of pharmaceutical innovation. In the two decades between 2005 and 2024, novel treatments for NTDs and other global health conditions accounted for just 2% of approvals by the U.S. Food and Drug Administration.²

To close this treatment gap and address unmet patient needs, we have harnessed our unique capabilities as an innovative medicines company to build one of the largest pipelines in the sector targeting NTDs.³ This is backed by more than USD 500 million investment in global health R&D since 2021.

Alongside our work on malaria, we work with trusted partners to:

•    discover, develop, and deliver treatments for NTDs such as dengue fever and leishmaniasis;
•    work on new candidates for conditions that are not formally classified as NTDs but continue to have a disproportionate impact on communities in low- and middle-income countries, including sickle cell disease and cryptosporidiosis; and
•    maintain strategic donation programs for diseases such as leprosy and parasitic worm infections, where we are the only remaining manufacturer and supplier of effective treatments.

Dengue fever

Dengue, also referred to as “break-bone fever” due to its intense muscle, joint and bone pain symptoms,⁴ is a viral disease transmitted to humans through the bite of infected mosquitoes. It is the most common vector-borne viral disease in the world,⁵ with about half of the world’s population now at risk,⁶ but there is currently no cure available.

We are developing the first potential antiviral for the treatment of dengue, which is currently in Phase II clinical trials.

Leishmaniasis and Chagas disease

We are working on multiple approaches to combat kinetoplast parasites, which cause devastating diseases such as leishmaniasis and Chagas disease.

There are an estimated 700 000 to 1 million new cases of leishmaniasis every year, transmitted through the bites of infected sandflies. It can be life-threatening in its visceral form. In its cutaneous form, it can cause disfiguring skin lesions and scarring.⁷

Chagas disease is transmitted by the “kissing bug” – so-called because it usually bites people’s faces while they sleep – and is a common cause of heart failure in endemic areas, often lying dormant for years or even decades. About 8 million people worldwide, mostly in Latin America, are estimated to be infected with the parasite that causes the disease.⁸

Together with partners such as the Drugs for Neglected Diseases Initiative, we are discovering and developing novel treatments for these diseases, including an oral antiparasitic compound currently in Phase II trials.

Leprosy and parasitic worms

Caused by a bacterial infection, leprosy is one of the world’s oldest diseases. Since the introduction of multidrug therapy in the 1980s, Novartis and partners have reduced cases by 96%,⁹ but there are still around 200 000 new cases reported every year.¹⁰

As the only remaining manufacturer, we have provided treatment for leprosy to the World Health Organization at no cost for more than 25 years, reaching more than 8.3 million people. We also donate treatments for fascioliasis and paragonimiasis, painful diseases caused by parasitic flatworms. In 2026, we renewed our partnership for a further five-year period.

Beyond this work, we are providing scientific and strategic advice to the TB Alliance on the development of a next-generation treatment for leprosy, which could represent the first innovation in treatment for more than 40 years.

Sickle cell disease

Sickle cell disease is an inherited blood disorder that affects almost 8 million people worldwide and is a major cause of death in children under the age of 5 in areas where it is highly endemic.¹² While treatment has advanced in recent decades, the disease often goes undiagnosed in low- and middle-income countries.¹³

Novartis is developing two new treatments, including Phase III trials for a monoclonal antibody designed to help reduce painful complications, and Phase II trials for an investigational small molecule therapy.

We also work with partners to improve diagnosis, expand access to quality care, and build stronger health systems for people living with sickle cell disease. In 2019 we helped to introduce sub-Saharan Africa’s first comprehensive sickle cell disease program in Ghana, which has since been adopted by eight countries in the region.

Cryptosporidiosis

Cryptosporidiosis is a major global cause of diarrheal disease, resulting in tens of millions of cases annually and causing substantial illness and death among children in lower-income countries.¹⁴ The greatest burden falls on children under five years of age, malnourished children, and immunocompromised individuals.¹⁵ Despite this substantial disease burden, treatment options remain limited.

Novartis is currently developing a direct-acting antiparasitic therapy for cryptosporidiosis that is being evaluated in a Phase II clinical trial.

Novartis has the industry’s #1 pipeline for malaria and NTDs, according to the Access to Medicine Foundation, and backed by more than USD 500 million investment since 2021.
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