Amicus jump starts launch of Fabry disease Therapy in UK

Amicus Therapeutics kicks off the commercial launch of its rare disease therapy, Galafold, in the UK after it was endorsed for reimbursement within the NHS by NICE’s Highly Specialised Technologies Evaluation Committee.

Galafold is a first-in-class chaperone therapy approved in the EU May last year as a monotherapy for the genetic disorder Fabry disease in patients with amenable mutations.

Fabry disease is an inherited lysosomal storage disease caused by a non-functional or only partially functional enzyme called alpha-galactosidase A (alpha-gal A), which leads to a build-up of enzyme substrates that cause cellular damage in tissues throughout the body. There are approximately 855 people with Fabry disease in England and currently there is no cure.

Amicus’ therapy is an oral, small molecule drug designed to bind to the enzyme as it is made, enabling helping it to fold correctly and improving its function. It is a life-long treatment, that costs £210,000 per patient per year without VAT and any discounts.

NICE concluded that in its final guidelines that by taking a confidential patient access scheme discount into account, Galafold has a lower total cost than enzyme replacement therapy (ERT), and can potentially provide greater health benefits.

However NICE also urged Amicus, NHS England and treatment centres to collect more evidence, especially with regards to 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.”

Source – PharmaTimes

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