Migalastat (Galafold): New treatment for Fabry disease

Last Updated on

January 12, 2018 – Fabry disease is an inherited disorder caused by mutations (alterations) in the alpha-galactosidase A (GLA) gene located on the X-chromosome. Fabry disease is rare and affects both males and females. It is estimated that classic Fabry disease (the most severe type) affects approximately one in 40,000 males. The later-onset type is more frequent, and in some populations, may occur in one in 1,500 to 4,000 males. Patients with Fabry disease develop slowly progressive kidney disease, cardiac hypertrophy (enlargement of the heart), arrhythmias (abnormal heart rhythm), stroke and early death. The genetic defect in GLA results from buildup of a type of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves and other organs

Until now, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease. In contrast, the American  Food and Drug Administration (FDA) recently approved Migalastat (Galafold), the first oral medication for the treatment of adults with Fabry disease. The drug is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive (“amenable”) to treatment with Migalastat (Galafold) based on laboratory data.

The efficacy of Migalastat (Galafold) was demonstrated in a six-month, placebo-controlled clinical trial in 45 adults with Fabry disease. In this trial, patients treated with Migalastat (Galafold) over six months had a greater reduction in globotriaosylceramide (GL-3) in blood vessels of the kidneys (as measured in kidney biopsy samples) as compared to patients on placebo.

The safety of Migalastat (Galafold) was studied in four clinical trials which included a total of 139 patients with Fabry disease. The most common adverse drug reactions in patients taking Galafold in clinical trials were headache, nasal and throat irritation (nasopharyngitis), urinary tract infection, nausea, and fever (pyrexia).

See here an explanation of Fabry disease:

Print Friendly, PDF & Email

Tags: , , , , , ,
About the Author
thassodotcom Ph.D.; Professor in Pharmacology and Toxicology. Senior expert in theragenomic and personalized medicine and individualized drug safety. Senior expert in pharmaco- and toxicogenetics. Senior expert in human safety of drugs, chemicals, environmental pollutants, and dietary ingredients.

Your opinion

Comment

No comments yet

thasso: conditions

thasso: tweets

thasso post: magazine

View my Flipboard Magazine.

thasso: categories

thasso: archives

thasso: simple chat

You must be a registered user to participate in this chat.

  • Genetics may help predict the right blood pressure drug for you June 25, 2019
    Medication can play a huge role in reducing high blood pressure, a leading cause of stroke, heart attack and other serious health problems. Yet given the wide selection of drugs for doctors to choose from, figuring out which drug works best for someone is difficult.
  • First in vivo proof-of-concept in Steinert's myotonic dystrophy June 25, 2019
    Ana Buj Bello's team, a researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has made the proof-of-concept of a CRISPR-Cas9 approach in a mouse model of Steinert's myotonic dystrophy, the most common neuromuscular disease in adults. Indeed, thanks to this genome editing approach, the expanded CTG triplet repeat in the DMPK gene, which […]
  • Gene networks reveal transition from healthy to failing heart June 24, 2019
    Scientists investigating heart failure have been limited to studying diseased heart tissue in the lab—understandably, as people don't tend to pluck out a healthy heart for the sake of research. But now, scientists with access to unusable, yet still healthy, donor hearts have been able to investigate the genomic pillars behind the transition from healthy […]
  • Microbiome links diet to health June 24, 2019
    The composition of the human microbiome, a complex ecosystem of microorganisms, plays a crucial role in lifelong health. Little is known, however, about the detailed molecular mechanisms linking health status to the microbiome of the gut, for example.
  • Researchers find new mutation in the leptin gene June 21, 2019
    The global obesity epidemic is so far-reaching it now has an overarching name: globesity. Texas Biomed Staff Scientist Raul Bastarrachea, M.D., is part of a team that discovered a new mutation in the gene that regulates the key hormone suppressing hunger called leptin. This new mutation could help researchers understand why people develop excess of […]
Top