Posted from: Monday, September 17, 2018 - 12:13 PM - Present

Galafold Approved to Treat Fabry Disease

August 10, 2018 – The U.S. FDA has approved Galafold™ (migalastat), manufactured by Amicus Therapeutics U.S., Inc., to treat Fabry disease in patients who are at least 16 years old and possess any of up to 348 galactosidase alpha (GLA) genetic variants found to be responsive to treatment with the drug. It is the first oral treatment for Fabry disease.

A rare but serious genetic disorder, Fabry disease can lead to progressive kidney disease, enlargement of the heart (cardiac hypertrophy), abnormal heart rhythms (arrhythmias), stroke, and premature death. It occurs when a genetic mutation causes inadequate production of alpha-galactosidase A (α-Gal A), an enzyme that breaks down a type of fat known as globotriaosylceramide (GL-3). The current standard of treatment has focused on enzyme replacement therapies. Galafold instead works by improving the activity of α-Gal A in the body. It is the first new therapy for Fabry’s disease to receive FDA approval in more than 15 years.

In a clinical study, patients who received Galafold for six months experienced a greater reduction of GL-3, as measured by kidney biopsy samples, than patients who received a placebo.

A clinical genetics professional should determine a patient’s suitability for treatment with Galafold based on GLA variants. Recommended dosing is one 123mg capsule taken once every other day at the same time on an empty stomach. Patients should not eat for at least two hours before and two hours after taking Galafold. The drug should not be taken on consecutive days.

Amicus launched the product immediately upon approval at a wholesale acquisition cost (WAC) of $315,000 per year.

Last Updated Friday, October 19, 2018 - 01:31 AM.