Pfizer has set up a new unit which will specifically focus on the treatment of diseases that affect fewer than 200,000 patients worldwide.
The Rare Diseases Research Unit will target the more-than-6000 orphan diseases, “as fewer than 10% have therapies that directly address” the underlying disorder, Pfizer says. Additionally, “the level of investment in rare disease research has traditionally been low, resulting in limited needed scientific advancements in this field”.
The new division will “pursue treatments across all therapeutic areas and modalities and will serve as the focal point for the company’s existing research on rare diseases”, Pfizer added. It also intends to “work closely with patient advocacy groups as it develops and advances the unit’s research strategy”.
Jose Carlos Gutierrez-Ramos, senior vice president of the New York-based giant’s Biotherapeutics R&D unit, said “we are coupling Pfizer’s existing experience in rare diseases, such as haemophilia, with our advanced protein technologies, resources and world-class scientific team to focus on becoming a driving force in rare disease research”. He added that the company has “a long history in discovering, developing and commercialising medicines that treat rare diseases and we are hopeful that this research unit will lead to additional new medicines for patients suffering from devastating illnesses for which there is no cure”.
The new unit got the thumbs-up from National Organization for Rare Disorders in the USA. Its chief executive, Peter Saltonstall, noted that “30 million Americans, 30 million Europeans and millions more around the world have rare diseases” and applauded the development of new treatments “for this medically-underserved population.”
The new unit will be lead by Edward Mascioli, most recently the head of Dapis Capital, a private equity firm. Previously he was vice president of clinical affairs at Peptimmune and senior medical director at Paraxel.
