Alseres Pharmaceuticals, Inc., announced that Cethrin has been granted designation as an orphan medicinal product for the treatment of traumatic spinal cord injury (SCI) by the European Commission. The European Commission's decision was adopted on September 5, 2008 following the favourable opinion issued by the European Medicine Agency (EMEA) Committee of Orphan Medicinal Products (COMP) at their meeting on June 10 and 11, 2008. The designation was granted to Triskel EU Services, Alseres' representative in the European Union (EU).
"The European orphan drug designation represents another important step for Alseres in the development of Cethrin," said James R. Weston, senior vice president of Regulatory Affairs and Quality. "We value the positive recognition from the EMEA's Committee on Orphan Medicinal Products of the potential for Cethrin to provided significant benefit to patients who experience a serious spinal cord injury."
Cethrin received Orphan Drug designation from the US Food and Drug Administration (FDA) in 2005 for the treatment of acute spinal cord injury. Currently in phase II clinical testing in the US and Canada, Cethrin is being investigated for its potential to restore motor and sensory function after acute spinal cord injury.
"Cethrin is one of only two drugs to ever receive Orphan Medicinal Product designation in the EU for the treatment of severe acute spinal cord injury. Having Orphan Designation in both the EU and US creates competitive advantages that further enhance Cethrin's value particularly because of the market exclusivity that comes with the designation," commented Dr. Mark Pykett, Alseres' president and chief operating officer.
Following drug approval, Orphan Designation provides a drug with market exclusivity for a 10 year period in the EU and for 7 years in the US for a particular indication. European Orphan Medicinal Product designations are based on various criteria that include: the life-threatening or debilitating nature of the condition, the lack of availability of other effective therapies, and the rarity of the condition. The European Union currently has 27 member countries, including France, Germany, Italy, Spain and the United Kingdom with a population of nearly half a billion people.
Cethrin is a recombinant protein drug that is being investigated for its potential to restore motor and sensory function after spinal cord injury. Following an SCI, about two-thirds of patients undergo decompression/stabilization surgery. During surgery, Cethrin is delivered by a single application to the injured region of the spinal cord.
A total of 48 patients with acute SCI were enrolled in a phase I/IIa open label study at nine sites across Canada and the United States. An escalating dose of Cethrin (0.3, 1, 3, 6 or 9 mg) was administered to the injured spinal cord during spinal decompression surgery. Neurological outcomes were measured using the ASIA Impairment Scale, or AIS. Evaluations were completed at 0, 1.5, 3, 6 and 12 months after treatment.
The data for the first 37 patients who completed the 12-month evaluation indicate that at 6 and 12 months after treatment, 38% (5/13) of cervical injury patients showed marked recovery of motor and sensory function after treatment as measured by a 2-grade or better improvement in the American Spinal Injury Association, or ASIA, Impairment Scale. A double-blind, randomized, placebo-controlled, multi-centre, phase IIb trial in patients with acute traumatic cervical SCI in the United States and Canada is expected to begin in 2009.
Alseres Pharmaceuticals, Inc. (ALSE) is engaged in the development of therapeutic and diagnostic products primarily for disorders in the central nervous system (CNS).