Amicus’ Galafold wins US approval

Doctors can now prescribe the drug for adults with the condition who have a genetic mutation determined to be responsive to treatment with Galafold based on lab data.

Fabry disease is a rare and serious genetic disease that results from buildup of a type of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves and other organs, caused by a non-functional or only partially functional enzyme called alpha-galactosidase A (alpha-gal A).

Patients develop slowly progressive kidney disease, cardiac hypertrophy, arrhythmias, stroke and early death.

Galafold is an oral, small molecule drug designed to bind to alpha-gal A as it is made, helping it to fold correctly and improving its function.

The drug’s efficacy was shown in a six-month, placebo-controlled clinical trial in 45 adults with Fabry disease, in which those treated with Galafold over six months had a greater reduction in GL-3 in blood vessels of the kidneys than patients given a placebo.

Galafold was approved using the Accelerated Approval pathway, which allows the US Food and Drug Administration to approve drugs for serious conditions where there is an unmet medical need if a treatment drug is shown to have certain effects reasonably likely to predict a clinical benefit.

A further study is needed to verify and describe the clinical benefits of Galafold in Fabry disease, the regulator noted.

"This FDA approval of Galafold is a transformative moment for people in the US living with Fabry disease, as it gives adult patients with amenable GLA variants a new treatment option for the first time in more than 15 years," noted chairman and chief executive John Crowley.

-------------------------------------------------------------------------------------------------------------------------------

Editor's Note:

To apply for becoming a contributor of en-CPhI.cn,

welcome to send your CV and sample works to us,

Email: Julia.Zhang@ubmsinoexpo.com.