NICE backs Amicus Therapeutics’ Galafold

The UK's NICE has recommended the use of Galafold (migalastat) on the NHS for the treatment of Fabry disease in draft Highly Specialised Technologies guidance.

The committee gave Amicus Therapeutics' genetic disorder treatment the provisional green light, bringing UK patients one step closer to an orally administered alternative to the standard fortnightly injections.

Despite initial uncertainty over its clinical effectiveness, the cost-effectiveness watchdog concluded that Galafold provides enough similar benefits to current enzyme replacement therapies (ERT), such as Sanofi's Fabrazyme (agalsidase beta) and Shire's Replagal (agalsidase alfa).

The regulator has agreed a confidential discount for the therapy with Amicus, creating a patient access scheme to reduce pricing from its annual treatment cost of £210,000 per patient in line with current ERT programmes.

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

An incurable inherited genetic disorder, Fabry disease is caused by a faulty enzyme responsible for breaking down fatty substances in the body's cells. Build up of lipids in blood vessel walls decreases blood flow to tissues and so often results in irreversible organ damage, progressive kidney and heart disease and increased risk of stroke.