Contact | 
Print version | 
PDF version | 
Send article | 
RSS-Feed
EMEA recommends Orphan Drug Designation for AX200 for the treatment of Amyotrophic Lateral Sclerosis
18 Feb 2008 -
SYGNIS Pharma AG announced that it has received a positive recommendation from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA) for AX200 for the treatment of Amyotrophic Lateral Sclerosis (ALS).
ALS is a progressive neurodegenerative disease caused by the degeneration of nerve cells innervating skeletal muscles and affects an estimated 50,000 to 100,000 people in the industrialized nations. Patients with ALS suffer from progressive muscle wasting throughout their bodies resulting in increasing disabilities and their lifespan is cut short. Death in ALS patients normally occurs within only a few years after diagnosis and there are currently no adequate therapies available.
The SYGNIS research team has demonstrated in pre-clinical trials that AX200 counteracts the neuronal cell death and thereby decelerates the development of the disease. In the laboratory, protection of the motor neurons led to a reduced degeneration of muscles whilst maintaining muscular strength for a longer period of time. Moreover, AX200 advances regenerative processes by stimulating neuronal stem cells. This dual mechanism of AX200 is a unique approach in the drug development for ALS.
Dr. Alfred Bach, CEO of SYGNIS Pharma AG, said: "Our pre-clinical data has convinced the EMEA experts that AX200 is a promising drug candidate for the treatment of ALS. The EMEA's positive opinion validates the great potential of AX200 for the treatment of such a difficult indication and supports us in our efforts to develop an effective therapy for ALS, which is one of the most devastating neurological diseases. We will plan further steps in close cooperation with EMEA and hope to initiate clinical trials within the next twelve months. Moreover, this is certainly an important signal for the future development of AX200 as a whole."
Top