SYGNIS Pharma AG announced that it has received Orphan Drug designation from the European Commission for AX200 in the treatment of spinal cord injury (SCI). This follows the positive recommendation that SYGNIS received from the EMEA in September.
Orphan Drug designation can be granted for a product that is intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union. During the development phase certain incentives are given to the developing company to facilitate the product's registration and market authorization. These incentives include reductions in fees, an accelerated registration procedure. Furthermore, for a period of up to 10 years market exclusivity is granted once the product is approved.
A spinal cord injury (SCI) is damage or trauma to the spinal cord which interrupts communication of the brain with body regions below the site of injury. Spinal cord injuries are predominantly caused by accidents and, in the majority of cases, result in life-long loss of control of motor functions and sensation. After the primary injury to the spinal cord, a cascade of events leads to progressive loss of tissue which may further deteriorate the patient's prognosis. There is a large demand for novel therapies in view of the poor treatment options currently available.
The SYGNIS research team has shown in pre-clinical trials that AX200 counteracts neuronal cell death. Furthermore, the compound has demonstrated a profound ability to support neuronal regeneration and neuronal plasticity which facilitates functional recovery after SCI.
In April 2008, SYGNIS received Orphan Drug Designation from the European Commission for AX200 in the treatment of Amyotrophic Lateral Sclerosis (ALS).
Dr Alfred Bach, CEO of SYGNIS Pharma AG, said, "We are very pleased to have also obtained the Orphan Drug status for AX200 for spinal cord injury. It is further validation of the potential of AX200 as a promising drug candidate in the treatment of diseases of the central nervous system. We look forward to exploiting this potential as we currently prepare a further phase-II efficacy study of AX200 in acute stroke."