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Amicus gets NICE green light for Fabry drug

Oral therapy alternative Galafold approved for routine NHS use

The National Institute of Health and Care Excellence has given its blessing to Amicus' oral therapy for Fabry disease, a rare genetic disorder.

The UK's cost-effectiveness regulator has published final guidance endorsing the use of Galafold (migalastat) for Fabry, an inherited lysosomal storage disease caused by deficiency in the enzyme alpha-galactosidase A (alpha-gal A).

Galafold is the first orally administered alternative to the current crop of injectable enzyme replacement therapies (ERT) for Fabry disease, such as Sanofi's Fabrazyme (agalsidase beta) and Shire's Replagal (agalsidase alfa), and works by stabilizing the body's own dysfunctional enzyme.

The drug was approved by the European Commission in May 2016, making its European debut in Germany in the same month, but Amicus suffered a setback in the US in November when the FDA asked for more data on the drug. It has also been approved in Switzerland.

In the US, Galafold has been recommended as an option for treating Fabry disease in patients aged over 16 with an amenable mutation and who would be candidates for ERT - on the condition that Amicus provides an undisclosed discount to the drug's list price of £210,000 per patient per year.

The final appraisal document from NICE notes that with the discount "migalastat has a lower total cost than ERT, and potentially provides greater health benefits than ERT," although it also notes there are gaps in the evidence for the drug, which should be plugged with further studies, particularly on long-term use.

Galafold is scheduled for its next review in 2020, but NICE said that could come sooner if the prices of ERTs change in the interim.

Fabry disease affects an estimated 855 people in England, of whom around 140 would be eligible for treatment with Galafold, according to Amicus.

At one point migalastat was licensed to GlaxoSmithKline (GSK), but the big pharma group returned rights to Amicus after a failed phase III trial in 2013.

Article by
Phil Taylor

22nd February 2017

From: Regulatory



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