MMV in partnership: the Eurartesim® experience.

BACKGROUND: This case study describes how a public-private partnership between Medicines for Malaria Venture (MMV) and Sigma-Tau Industrie Farmaceutiche Riunite SpA achieved international regulatory approval for use of the fixed-dose artemisinin-based combination therapy dihydroartemisinin-piperaquine (Eurartesim®) for the treatment of malaria, enabling more widespread access to the medicine in malaria-endemic countries. CASE DESCRIPTION: The combination of dihydroartemisinin and piperaquine demonstrated success in clinical trials for the treatment of malaria in Asia and Africa in the 2000s. However, as it had not been developed to international regulatory standards it was out of the reach of the majority of patients in disease-endemic countries, particularly those reliant on public healthcare systems supported by international donor funding. To overcome this, as of 2004 MMV worked in partnership with Sigma-Tau, Holleykin, Oxford University, the Institute of Tropical Medicine Antwerp, and the National Institute of Malaria Research India to develop the dihydroartemisinin-piperaquine combination to international standards. In 2011, the European Commission granted full marketing authorization to Sigma-Tau for Eurartesim. DISCUSSION AND EVALUATION: The partnership between MMV, Sigma-Tau, and numerous other academic and industrial partners across the world, led to the successful development to EMA regulatory standards of a high-quality and highly efficacious anti-malarial treatment that otherwise would not have been possible. The dossier has also been submitted to the WHO for prequalification, and a safety statement to guide correct use of Eurartesim has been produced. In July 2012, the first delivery to a disease-endemic country was made to Cambodia, where the medicine is being used to treat patients and help counter the emergence of artemisinin resistance in the area. A paediatric dispersible formulation of Eurartesim is being developed, with the objective to submit the dossier to the EMA by the end of 2014. CONCLUSIONS: The development of Eurartesim to international regulatory standards exemplifies the strengths of the product development partnership model in utilising the individual skills and expertise of partners with differing objectives to achieve a common goal. Successful uptake of Eurartesim by public health systems in malaria-endemic countries poses new challenges, which may require additional partnerships as we move forward.

When is enough enough? The need for a robust pipeline of high-quality antimalarials.

Malaria kills an estimated one million people a year--mostly children under 5. State-of-the-art medicines known as artemisinin combination therapies (ACTs) are available, and successfully cure up to 99% of patients. Additionally, insecticide-treated bed nets and insecticide spraying are helping to prevent the disease, while a vaccine is in clinical development. With all these tools at hand, you might ask why we need more new medicines. Why not just concentrate on improving the distribution of existing ones? Whilst access to medicines is clearly a major challenge, there are several reasons why new antimalarials are urgently needed--and will continue to be needed until we have finally defeated the parasite. First, the emergence of drug resistance to any infectious disease treatment is inevitable. A range of medicines with varying mechanisms of action are needed to stem the tide of drug resistance as well as fill the gap when it takes hold. Second, malaria is a disease predominantly affecting children and expectant mothers. These vulnerable patient groups require medicines tailored to their needs with robust safety profiles. Third, of the five species of malarial parasites that infect humans, two can relapse, and there is currently no safe medicine to combat the relapse for all patients. Finally, in order to ultimately eradicate malaria, medicines are needed that go a step beyond simple treatment and break the transmission of the parasite from patient to patient.