UK launch for first oral drug in Fabry disease

28 February 2017

US biotech firm Amicus Therapeutics (Nasdaq: FOLD) has started the commercial launch of the precision medicine Galafold (migalastat) in the UK.

This follows the final publication of reimbursement guidelines by the National Institute for Health and Care Excellence (NICE), recommending that Galafold be reimbursed within the National Health Service (NHS) in England as a first-line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.

John Crowley, chief executive of Amicus Therapeutics, said: “Galafold is the first oral treatment as well as the first precision medicine now available in the UK for Fabry patients 16 years and older who have an amenable mutation.

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