Case Study: Research and Development

DNDi Partnership for Development of an Oral Treatment of Sleeping Sickness, Sanofi

Objectives

Transmitted by the bite of tsetse flies, Human African Trypanosomiasis, also known as sleeping sickness, is one of the tropical diseases affecting the poorest populations of remote rural areas of Africa. When left untreated, the disease is always fatal. There is, currently, no simple treatment for this disease. Available therapeutic options are either poorly tolerated or difficult to administer since they require intravenous infusions. This is why Sanofi, in partnership with the non-profit Drugs for Neglected Diseases initiative (DNDi), signed in 2009 an agreement for the development, manufacturing and distribution of fexinidazole, a promising new oral treatment. Sanofi and DNDi have decided to combine their expertise to develop and make fexinidazole available on a non-profit basis to all patients who need it. Under the terms of the agreement, DNDi is responsible for preclinical, clinical and pharmaceutical development. Sanofi is responsible for the industrial development, registration, and production of the drug at its manufacturing sites.

Lessons Learned

The project is in the direct line of the previous success of Sanofi’s first collaboration with DNDi on the development of a drug for the treatment of malaria which naturally led to the expansion of the partnership. It demonstrated how innovative models for drug development can be implemented to address neglected diseases.

Results to Date

  • Clinical development programme now in phase II/III
  • Development of dry blood (and cerebroaspinal fluid, CSF) spot testing for fexinidazole, which eliminates need for storage at low temperature of blood and CSF samples taken from patients
  • Finalisation of industrial API process by Sanofi at their Budapest development centre.
  • Regulatory strategy under discussion in close collaboration with the WHO, the European Medicines Agency and the US Food and Drug Administration.

Fexinidazole was advanced all the way from discovery into clinical development, and is currently the only compound in clinical development for the treatment of sleeping sickness. Because it would greatly simplify treatment of sleeping sickness, this project holds great promise for the patients and the practitioners in the field.