The European Medicines Agency, following a record year for orphan drug designations, has already taken steps toward naming the first orphan medicines of 2010 with the recommendation of four to the European Commission.
In 2009, the commission granted orphan status to 103 medicines, the highest since Europe's orphan medicines legislation was introduced in 2000. Annual applications to the EMEA, and subsequent orphan designations by the commission, have climbed fairly steadily since, according to the commission's monthly report.
Europe's orphan medicines legislation, modeled in part on a much older law in the United States, was designed to encourage the development of medicines to treat diseases affecting fewer than five in 10,000 E.U. residents, medicines that pharmaceutical companies previously had little financial incentive to pursue. The E.U. law provides reductions in fees for regulatory procedures and a minimum 10-year monopoly on any orphan drug granted marketing approval.
In 2007, the U.S. Food and Drug Administration and the European Medicines Agency introduced a common application for orphan drug status, making it easier for pharmaceutical firms to apply for orphan medicine status in both Europe and the United States, though each government retains separate criteria, separate approval processes, and different forms of financial assistance to pharmaceutical firms testing orphan drugs.
The common application may have helped increase the number of recent bids for orphan status in Europe, said EMEA spokeswoman Monika Benstetter, though, she added, the law's built-in financial incentives are probably sufficient to explain the annual increases since 2000.
"Cooperation with FDA is of course meant to make it easier for small and medium-size enterprises that are the main business drivers of orphan medicine development," Ms. Benstetter said. "It certainly has had an impact but we have not quantified that impact."
When EMEA's orphan medicines committee met Jan. 5 and 6, during its first meeting of the new year, the committee recommended that the European Commission consider granting orphan status to four medicines: An unnamed compound for the treatment of acute myeloid leukemia (Merck); davunetide for progressive supranuclear palsy (FGK Representatitve Service); lentiviral vector containing the human MYO7A gene for retinitis pigmentosa in Usher syndrome 1B (Oxford Biomedica), and taliglucerase alfa for Gaucher disease (Protalix B.V.).
Six other medicines have been granted orphan status by the European Commission since the orphan medicine committee's December 2009 meeting.
[Editor's note: This story appears courtesy of Elsevier Global Medical News. For more information, please contact Michael Magoulias at 240-221-4530 .]
- Jennie Smith