Pfizer forms rare diseases unit
pharmafile | June 15, 2010 | News story | Research and Development | Orphan, Pfizer
Pfizer is seeking to boost its coverage of research into orphan conditions with the creation of a new unit focused on rare diseases.
Orphan conditions are an increasingly attractive target for pharma companies looking for new commercial opportunities, with the company’s rival GlaxoSmithKline launching a similar specialist unit earlier this year.
These moves make sense as more than 6,000 diseases are classified as ‘orphans’ but fewer than 10% have therapies that directly address their underlying causes.
Pfizer’s aim is to find novel medicines for diseases affecting fewer than 200,000 patients, explained Jose Carlos Gutierrez-Ramos, senior vice president, biotherapeutics research and development.
“We are coupling Pfizer’s existing experience in rare diseases, such as haemophilia, with our advanced protein technologies, resources and world-class scientific team,” he said.
The unit is led by Edward Mascioli, who will be based in Cambridge, Massachusetts and will report to Gutierrez-Ramos.
Mascioli is the founder of Dapis Capital, a private equity firm focused on life sciences, and was previously vice president of clinical affairs at Peptimmune and senior medical director at Paraxel.
The US National Organization for Rare Disorders (NORD) estimates that 30 million Americans, 30 million Europeans and millions more around the world suffer from a rare disease, and most of which have no available treatment.
Many of the diseases are genetic in origin, start in childhood and can cause lifelong disability and premature death.
“NORD applauds Pfizer’s commitment to expanding its research with the goal of development new treatments for this medically underserved population,” said NORD president Peter Saltonstall.
One of the problems has been that the level of investment in rare disease research has traditionally been low, which means scientific advances have been limited.
Pfizer’s unit will look at treatments across all therapeutic areas and the company promises to work closely with patient advocacy groups.
“Pfizer 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,” said Gutierrez-Ramos.
Adam Hill
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