Orphan drug launches for rare diseases reached a 10-year high in 2011, underscored by new breakthroughs in personalized medicine, according to a report by IMS Institute for Healthcare Informatics in New Jersey.
Orphan drugs carry a higher price tag than other drugs because they are intended for a patient population of no more than 200,000.
Among the 12 orphan drugs launched last year was Pfizer’s (NYSE:PFE) drug critzotinib, branded Xalkori, to treat nonsmall cell lung cancer. The drug targets a specific biomarker — the anaplastic lymphoma kinase fusion gene. The therapy is for 3 percent to 7 percent of patients with tumors that carry a unique mutation and can improve survival by nine months. Roche-owned Genentech drug, sumotropin, a human growth hormone, was launched to alleviate the short stature associated with Turner syndrome, a disorder that affects women in which they lack or have a malfunctioning sex chromosome.
http://www.medcitynews.com/2012/04/orphan-drug-launches-reach-10-year-high-in-2011/?edition=pharmaceuticals
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