ALS is a severe, neurodegenerative disease which affects motor neurons leading to progressive muscle weakness, paralysis and ultimately death within a median time of two to four years from disease onset.
According to the firm, the ODD recognises the potential therapeutic role of aldesleukin in treating disease, and could provide a number of benefits for Clinigen should it obtain a marketing approval for this indication.
These benefits include seven years marketing exclusivity within the US upon launch, along with tax credits for clinical development costs and fee waivers.
Studies in ALS patients have shown that decreased levels of regulatory T-cells (Tregs) were associated with increased disease severity and predictive of disease progression and survival, suggesting that Tregs may be an attractive therapeutic target.
http://www.pharmatimes.com/news/clinigens_als_therapy_bags_us_orphan_status_1344779