3 January 2017 - England and Wales’ cost effectiveness watchdog NICE has given a final recommendation to a new treatment for the rare Fabry disease.
Amicus Therapeutics’ Galafold (migalastat hydrochloride) is the first oral treatment for the disease, and represents a rival to established enzyme replacement therapies, namely Genzyme’s Fabrazyme and Shire’s Replagal.
The treatment could now gain significant market share from the existing treatments, especially as NICE says it represents better value for money, and may also provide more clinical benefits.