Migalastat

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Migalastat Brand Name– Galafold

What is Migalastat

Migalastat is an oral alpha-galactosidase A (alpha-Gal A) pharmacological chaperone indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay.

Fabry disease is a rare, X-linked lysosomal storage disorder caused by mutations in the GLA gene encoding the enzyme alpha-Gal A.

Complete or partial deficiency in this enzyme leads to intracellular accumulation of the glycosphingolipids, globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3).

Glycosphingolipids accumulate in the lining of blood vessels in the heart, kidney, and other organs in patients without an adequate presence of alpha-Gal A. Crises of severe pain in the extremities (acroparesthesias), hypohidrosis, corneal opacities, and dysfunction of several organs (kidney, brain, heart) are the primary manifestations, and patients often have decreased life expectancy and experience renal failure, cardiomyopathy, and cerebrovascular accidents.

A correct diagnosis of Fabry disease has considerable implications regarding treatment, management, and counseling.

Prior to migalastat treatment, the amenable GLA variant must be interpreted by a clinical genetics professional as causing Fabry disease (pathogenic, likely pathogenic) in the clinical context of the patient. In cases where the amenable GLA variant is of uncertain clinical significance (VUS, variant of uncertain significance) or may be benign (not causing Fabry disease), consultation with a clinical genetics professional is strongly recommended.

In a clinical trial of 45 adults with Fabry disease, patients treated with migalastat for 6 months had a greater reduction in GL-3 in blood vessels of the kidneys, measured by biopsy samples, compared to patients receiving placebo.

Migalastat is well tolerated with the most common adverse drug reactions being headache, nasopharyngitis, urinary tract infection, nausea, and fever.

Indications

  • Fabry disease

For the treatment of Fabry disease in adults with an amenable galactosidase alpha gene (GLA) variant

NOTE: Migalastat has been designated as an orphan drug for this indication by the FDA

Side Effects

  1. abdominal pain
  2. back pain
  3. cough
  4. cystitis
  5. diarrhea
  6. epistaxis
  7. fever
  8. headache
  9. infection
  10. nausea
  11. pharyngitis
  12. vomiting

Adverse reactions reported in at least 5% of migalastat-treated patients (n = 34) in a 6-month, placebo-controlled trial included

  • headache (35%)
  • naso-pharyngitis (18%)
  • urinary tract infection (urinary tract infection, cystitis, kidney infection
  • fever
  • back pain (9%)
  • cough (9%)
  • epistaxis (9%)

Monitoring Parameters

  • laboratory monitoring not necessary

Contraindications

  • breast-feeding
  • dialysis
  • infertility
  • pregnancy
  • renal failure
  • renal impairment

Interactions

There are no drug interactions associated with Migalastat products.

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