Pfizer 2011 Annual Review | Pfizer: the world's largest research-based pharmaceutical company

Treating Neglected and Rare Diseases

We see both opportunity and purpose in working to eradicate diseases of the developing world and to find effective treatments for diseases that are devastating but may affect only a very few.

Pfizer has a long history of contributions to research aimed at controlling or eliminating diseases that disproportionately impact poor patients in the developing world. We are currently working to address malaria in pregnancy, the most common and yet preventable cause of maternal and perinatal morbidity and mortality in sub-Saharan Africa. About 125 million pregnancies are at risk of malaria every year and 10,000 women and up to 200,000 babies die as a result. The emergence of resistance to sulfadoxine-pyrimethamine, the currently adopted Intermittent Preventative Treatment in Pregnant Women (IPTp), has prompted a need to develop new and effective regimens. Pfizer and Medicines for Malaria Venture are working together to develop a fixed-dose combination tablet of azithromycin and chloroquine for IPTp. This program is currently in Phase III of clinical development. More information on Pfizer's malaria efforts in sub-Saharan Africa is available on our website. Pfizer also provides a variety of treatments, and seeks to develop new ones, for HIV/AIDS, through our joint venture with GlaxoSmithKline, known as ViiV Healthcare.

WIPO Re:Search: Sharing Innovation in the Fight Against Neglected Tropical Diseases

In partnership with the World Intellectual Property Organization (WIPO) and BIO Ventures for Global Health (BVGH), Pfizer and the R&D pharmaceutical industry launched an R&D consortium, known as WIPO Re:Search, in October 2011. The consortium was created to accelerate the discovery and product development of medicines, vaccines and diagnostics to develop new solutions for neglected tropical diseases, malaria and tuberculosis. This work is unprecedented as it includes diverse global health partners such as multilateral organizations, nongovernmental organizations and patent offices, as well as private and public research organizations from around the world, including the National Institutes of Health, Medical Research Council (South Africa), Drugs for Neglected Diseases initiative, Fundação Oswaldo Cruz, Indian Council for Medical Research, and the U.S. Patent and Trademark Office.

The consortium addresses several factors that have previously impeded the development of effective solutions including inadequate networks for sharing information, the need for a central information point where prospective partners can learn of ongoing work and capabilities in specific diseases, cumbersome intellectual property licensing procedures, and inadequate funding to support vibrant and consistent R&D. When fully in place, WIPO Re:Search will provide three services:

  • A comprehensive platform/database, hosted by WIPO, of patent and other proprietary information (such as clinical trial results) on compounds and technologies available for licensing for neglected tropical diseases research.
  • A partnership hub, managed by BVGH, providing a forum where interested parties can learn about licensing opportunities, available funds for research and networking opportunities within their respective fields.
  • A range of specific supporting activities to increase and improve licensing agreements and to quickly resolve disputes over intellectual property.

Pfizer's contributions to WIPO Re:Search include patents for veterinary agents that might provide clues to more effective treatment of human worm infections and intellectual property covering selected vaccine technologies. In addition, we are sharing information from our animal health research that has the potential to be leveraged for human neglected tropical parasitic diseases.

For more information, please visit the WIPO Re:Search website, available at

Rare Diseases

Rare diseases affect millions of patients and their caregivers around the world—yet fewer than five percent of the estimated 7,000 rare diseases have approved treatments. Pfizer is dedicated to addressing these unmet medical needs by seeking to discover, develop and deliver medicines for them. Currently our pipeline includes a number of medicines in development for rare diseases, including Gaucher disease and complications of sickle cell anemia.

2011 Highlights

  • A novel collaborative research agreement between Children's Hospital Boston and Pfizer is directed to the development of new therapies for Duchenne muscular dystrophy. We are providing access to select proprietary compounds that will be tested in the hospital's world-class academic research laboratory, and committing Pfizer resources, such as expertise in medicinal chemistry, to this cause.
  • GlycoMimetics, Inc. and Pfizer entered into an exclusive worldwide licensing agreement for a drug candidate, GMI-1070, currently in development to treat patients experiencing vaso-occlusive crisis associated with sickle cell disease. GMI-1070 has received Orphan Drug and Fast Track status from the U.S. Food and Drug Administration. While the genetic and molecular cause of sickle cell disease has been known for more than 50 years, therapy has not significantly advanced.
  • Vyndaqel (tafamidis) was approved in the EU in November 2011 for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) in adult patients with Stage 1 symptomatic polyneuropathy. This approval represents a major advance as Vyndaqel is the first and only medication approved to delay neurologic impairment for patients suffering from this rare, progressive and nearly always fatal neurodegenerative disease, estimated to affect 8,000 people worldwide.


For more information on Pfizer's Malaria Efforts, view Malaria Efforts.