Amicus Therapeutics has kicked off the commercial launch of its rare disease therapy Galafold in the UK after it was endorsed for reimbursement within the NHS by the National Institute for Health and Care Excellence's Highly Specialised Technologies Evaluation Committee.
Galafold (migalastat) is a first-in-class chaperone therapy approved in the European Union in May last year as a monotherapy for the genetic disorder Fabry disease in patients with amenable mutations.
Fabry disease an inherited lysosomal storage disease caused by a non-functional or only partially functional enzyme called alpha-galactosidase A (alpha-gal A), which results in the build up of enzyme substrates that cause cellular damage in tissues throughout the body and for which there is no cure.
Galafold is an oral, small molecule drug designed to bind to this enzyme as it is made, helping it to fold correctly and improving its function. It is a life-long treatment, that costs £210,000 per patient per year (excluding VAT and any discounts).
In final guidelines, NICE concluded that, when taking a confidential patient access scheme discount into account, Galafold has a lower total cost than enzyme replacement therapy (ERT), and potentially provides greater health benefits.
But it also urged Amicus, NHS England and treatment centres to collect more evidence, particularly on the longer-term benefits of the therapy and ERT for treating Fabry disease, which should inform a future evaluation of the costs and benefits of all treatment options for the condition.
"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," stated John F. Crowley, chairman and chief executive of Amicus.
"The finalisation of pricing and reimbursement in the UK is a significant milestone for Amicus as we navigate the country-by-country processes to launch Galafold throughout the EU as rapidly as possible."
It is estimated that there are 855 people with Fabry disease in England.