Sygnis ALS treatment gets orphan status
German Sygnis Pharmaceuticals' amyotrophic lateral sclerosis treatment, AX200 has been granted an Orphan Drug designation by the European Commission and the firm intends to begin clinical trials within the next 12 months. This follows a recommendation from the European Medicines Agency (EMEA, Marketletter February 25).
ALS is a progressive neurodegenerative disease caused by the degeneration of nerve cells. It affects an estimated 50,000 to 100,000 people in industrialized nations.
The AX200 drug candidate has a dual mechanism. Firstly, it counteracts neuronal cell death, decelerating the slowing of the disease. Secondly, it stimulates neuronal stem cells, advancing regenerative processes.
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