Cost, functionality, adverse effects, operation mechanism, and additional details about Galafold
Galafold, a prescription medication, has been approved by the FDA to treat Fabry disease in adults with a specific gene change called galactosidase alpha (GLA). This oral treatment, marketed as migalastat, was first approved in 2018 and has demonstrated sustained commercial growth, indicating ongoing patient use and acceptance [1][4].
Fabry disease is a rare, inherited condition that causes fat to build up throughout the body, particularly in the kidneys, nervous system, and cardiovascular system. It is caused by a deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A) [2]. Galafold works by stabilizing and enhancing residual enzyme activity in patients with amenable mutations, potentially slowing disease progression [3].
The recommended dosage for Galafold is 123 mg taken once every other day. It is meant to be used as a long-term treatment for Fabry disease. Missed doses should be taken as soon as remembered, but if it has been more than 12 hours since the regularly scheduled time, the missed dose should be skipped and the next scheduled dose taken at the usual time [4].
While the effectiveness of Galafold is based on early clinical trials, the relationship between reducing GL-3 lipid buildups and relief of Fabry disease symptoms has not been proven [5]. Mild side effects of Galafold can include headache, common cold, mild urinary tract infection (UTI), nausea, fever, pain in the abdomen, back pain, cough, diarrhea, nosebleed, and vomiting [4]. Allergic reactions to Galafold are possible, although they weren't reported in clinical studies. Symptoms of a mild allergic reaction include skin rash, itchiness, and flushing (temporary warmth, redness, or deepening of skin color). A more severe allergic reaction is rare but can include swelling under your skin, swelling of the tongue, mouth, or throat, and trouble breathing [4].
Financial and insurance assistance is available for Galafold through the manufacturer's program called Amicus Assist. However, before approving coverage for Galafold, insurance companies may require prior authorization [4]. Galafold comes as a capsule that is taken orally and is available in one strength: 123 milligrams (mg) [4].
It is not known if Galafold is safe to take while pregnant or breastfeeding. Pregnant or breastfeeding individuals should talk with their doctor before taking Galafold [4]. Galafold isn't currently known to interact with other medications, herbs, supplements, or foods. However, before taking Galafold, you should still talk with your doctor and pharmacist about all prescription, over-the-counter, and other drugs you take, as well as any vitamins, herbs, and supplements you use [4].
Galafold works by decreasing the number of globotriaosylceramide (GL-3) buildups as measured by a kidney biopsy, which may help ease symptoms of Fabry disease. However, the relationship between reducing GL-3 lipid buildups and the easing of Fabry disease symptoms hasn't been proven [3]. If you develop symptoms of a mild or severe UTI while taking Galafold, talk with your doctor right away [4]. You should not crush, open, or chew Galafold capsules. You should swallow them whole [4].
For comprehensive clinical insights on long-term effects such as organ function preservation, quality of life, or adverse effects, consulting recent clinical trial results or Fabry disease treatment guidelines would be necessary beyond the current summary [5].
[1] https://www.reuters.com/article/us-amicus-results-idUSKBN1ZI2C2 [2] https://ghr.nlm.nih.gov/condition/fabry-disease [3] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5663325/ [4] https://www.galafold.com/ [5] https://www.drugs.com/galafold.html
- Further research in the field of genetics, specifically on other genetic mutations related to Fabry disease, could potentially lead to the development of additional treatments like Galafold for health-and-wellness.
- In addition to managing medical-conditions like Fabry disease, maintaining a dialogue with healthcare professionals about prescription medications such as Galafold and their potential interactions with other drugs, herbs, supplements, or foods, is essential for overall health-and-wellness.
- Pharmacies should be aware of insurance companies' requirements for prior authorization when dealing with prescriptions for medications such as Galafold, as there may be financial assistance programs available through the manufacturer for medical-conditions like Fabry disease.
