Please login to the form below

Not currently logged in
Email:
Password:

NICE backs Amicus Therapeutics’ Galafold

Recommends oral alternative for treating Fabry disease
NICE

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.

Article by
Rebecca Clifford

28th October 2016

From: Regulatory

Share

Tags

PMEA Awards 2020

COVID-19 Updates and Daily News

Featured jobs

PMHub

Add my company
Madmicrobe Studios

MadMicrobe is an award-winning team of certified medical illustrators creating animations that draw audiences into a world of suspense, excitement,...

Latest intelligence

#DemandDiversity: For International Women's Day, we ask... why do women often suffer from more side effects than men?
Women are largely prescribed exactly the same treatment regimens as men, with no account for the underlying differences in physiology and drug metabolism between the sexes....
Good design saves lives
Good design and creative thinking are essential if we are to improve on existing problems in new ways, which is why design and creativity within healthcare is vital. Health is...
Why you must understand the pricing of patient recruitment companies
Recruiting a diverse range of patients and engaging with them for your clinical trial isn’t an easy task, which means you might turn to patient recruitment companies, like us, who...

Infographics