When was Galafold approved?
Development Timeline for Galafold
Date | Article |
---|---|
Aug 11, 2018 | Approval FDA Approves Galafold (migalastat) for the Treatment of Fabry Disease |
Feb 12, 2018 | U.S. FDA Files New Drug Application Under Priority Review for Migalastat for Treatment of Fabry Disease |
Where is Galafold approved?
Galafold is approved in over 40 countries around the world, including the U.S., EU, U.K., Japan and others. Galafold is 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 data.
What is Galafold used for?
GALAFOLD is a prescription medicine used to treat adults with Fabry disease who have a certain genetic change (variant) in the galactosidase alpha gene (GLA) that is responsive (amenable) to GALAFOLD.
Is Galafold an enzyme replacement therapy?
Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research.
How many patients have Fabry?
Fabry disease is a rare pan-ethnic disorder, meaning that it occurs in all racial and ethnic populations affecting males and females. It is estimated that type 1 classic Fabry disease affects approximately one in 40,000 males.
How is Fabry disease treated?
Enzyme replacement therapy (ERT) is the only specific treatment for Fabry disease. The beneficial effect of ERT on different organs/systems has been extensively evaluated. Quality of life of patients receiving ERT is improved.
What is Fabry disease symptoms?
Fabry disease symptoms include:
- Numbness, tingling, burning or pain in the hands or feet.
- Extreme pain during physical activity.
- Heat or cold intolerance.
- Abnormal opacity of the eye (cornea), which does not change someone’s vision.
- Dizziness.
- Flu-like symptoms, including fatigue, fever and body aches.
Is Galafold a gene therapy?
Enzyme replacement therapy (ERT) with recombinant human alpha-gal A or gene-activated alpha-gal A is the current standard of care for these patients. Galafold is what scientists call a chaperone therapy, and is the first of this class of medications.
Can Fabry cause hair loss?
Other symptoms that may be associated with Fabry disease include chronic fatigue, dizziness, headache, generalized weakness, nausea, and/or vomiting, delayed puberty, lack of or sparse hair growth, and rarely malformation of the joints of the fingers.
What is the life expectancy of someone with Fabry disease?
The life expectancy of males with Fabry disease was 58.2 years, compared with 74.7 years in the general population of the United States. The life expectancy of females with Fabry disease was 75.4 years, compared with 80.0 years in the United States general population.
How is Fabry diagnosed?
How is Fabry disease diagnosed?
- Enzyme assay: This test measures alpha-GAL enzymes in blood.
- Genetic: Because females with Fabry disease can have normal levels of alpha-GAL enzymes, providers use genetic testing (DNA sequencing) to identify the GLA gene mutation.
When to take Migalastat for Fabry disease?
Oral migalastat is the first pharmacological chaperone approved for treating patients [aged ≥ 18 years (USA and Canada) or ≥ 16 years in other countries] with Fabry disease who have a migalastat-amenable GLA mutation.
How big is a 2 mg Migalastat capsule?
Capsules: 123 mg of migalastat in a size “2” capsule with an opaque blue cap and opaque white body with “A1001” printed in black, containing white to pale brown powder. 2 Reference ID: 4305282 4
When was Galafold first approved by the FDA?
FDA Approved: Yes (First approved August 10, 2018) Brand name: Galafold. Generic name: migalastat. Dosage form: Capsules. Company: Amicus Therapeutics.
When to consult with a glacologist about GLA?
Consultation with a clinical genetics professional is strongly recommended 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).