Migalastat for Fabry Disease Shows Promise

By Reuters Staff

April 30, 2014

(Reuters) - Amicus Therapeutics Inc today announced positive 12- and 24-month data from a phase 3 study of its experimental drug migalastat as monotherapy for Fabry disease.

Fabry disease is an inherited, potentially fatal disorder characterized by the buildup of a particular type of fat - most notably in the kidneys - called globotriaosylceramide, or GL-3, in the body's cells.

This progressive lipid accumulation, caused by the deficiency of the enzyme -galactosidase A (-Gal A), results in cell damage, leading to pain, hearing loss, kidney failure, heart attacks and strokes. As a monotherapy, migalastat works by binding to the -Gal A enzyme, made in the patient's body, helping it break down the lipids.

The company said subjects who switched from placebo to migalastat after month six demonstrated a statistically significant reduction in kidney interstitial capillary GL-3 at month 12 (p=0.013).

Also, subjects who remained on migalastat for 12 months demonstrated a durable reduction in kidney interstitial capillary GL-3.

A reduction in disease substrate was also observed in plasma lyso-Gb3, another important biomarker of disease, in subjects who switched from placebo to migalastat (p<0.0001), the company reported. Subjects who remained on migalastat demonstrated a durable reduction in lyso-Gb3.

Kidney function remained stable over 18-24 months and migalastat was generally safe and well-tolerated, the company said.

In a statement, John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, said: "We are pleased to report that the 12 and 24 month results from Study 011 have met our pre-defined criteria for success in terms of substrate reduction at 12 months, as well as clinical measures of kidney function maintained out to 24 months. We believe these data provide important validation that a small-molecule chaperone can restore the function of a patient's own enzyme in patients with amenable mutations, and that our pharmacogenomic assays can identify these patients."

Cowen & Co analyst Edward Nash said migalastat could satisfy the "significant need for an oral therapy for Fabry disease".

Sanofi SA's Fabrazyme, the first FDA-approved Fabry treatment, is administered intravenously.

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