Galafold, with the generic name migalastat, stands as a beacon of innovation in the realm of precision medicine. It offers a unique approach to treating a specific group of individuals with a rare genetic disorder. This blog explores the various facets of Galafold, from its manufacturing details to FDA approval, highlighting its significance in the medical landscape.
Manufacturer and FDA approval
Manufactured by Amicus Therapeutics, Galafold undergoes a meticulous production process adhering to stringent quality standards. This commitment to excellence ensures a reliable and effective treatment option for individuals in need. Galafold has earned approval from the U.S. Food and Drug Administration (FDA) on August 10, 2018, for its safety and efficacy, underscoring its credibility as a therapeutic intervention.
What is Galafold?
Galafold (migalastat) is an alpha-galactosidase A (alpha-Gal A) which belongs to the class known as pharmacological chaperone. This medicine is designed for treating adults with a confirmed diagnosis of Fabry disease and possessing a galactosidase alpha gene variant amenable to intervention. Galafold is the first and only oral therapy for adults with Fabry disease and an amenable GLA variant.
What is Fabry Disease?
Fabry disease is a rare genetic disorder that is caused by a mutation in the GLA gene. The GLA gene provides instructions for making the alpha-galactosidase A (GLA) enzyme. GLA is responsible for breaking down a fatty substance called globotriaosylceramide (Gb3). When GLA is defective, Gb3 accumulates in cells throughout the body, causing damage and disease. Fabry disease can affect both males and females, but it is more severe in males.
Symptoms Of Fabry Disease
Symptoms can begin in childhood or early adulthood. Some of the most common symptoms include:
- Painful burning or tingling sensations in the hands and feet
- Skin rash
- Kidney problems
- Heart problems
- Stroke
How Does Galafold Work?
Galafold is a chaperone therapy, which means that it works by helping a defective enzyme to fold properly and function normally. The defective enzyme in Fabry disease is GLA.
Galafold works by stabilizing the GLA enzyme and helping it to fold properly. This allows GLA to function normally and break down Gb3. As a result, Galafold can help to improve symptoms and slow the progression of Fabry disease.
Benefits of Galafold
Galafold has been shown to be effective in slowing the progression of Fabry disease and improving the quality of life for patients. Some of the benefits of Galafold include:
- Reduced pain and burning sensations in the hands and feet
- Improved kidney function
- Reduced risk of heart problems and stroke
- Improved quality of life
- Side Effects of Galafold
Recommended Dosage
Galafold’s dosage is tailored to the individual’s weight, ensuring optimal effectiveness. Adhering to the prescribed dosage is essential for a successful treatment outcome.
- For adults with confirmed Fabry disease and an amenable GLA variant, administer GALAFOLD based on clinical genetics professional interpretation.
- The recommended dosage is 123 mg orally every other day on an empty stomach, with a 4-hour fasting window.
- Avoid consecutive daily doses, and if a dose is missed within 12 hours, take it; otherwise, resume on the next planned dosing day.
- Swallow capsules whole; do not cut, crush, or chew. Consult a genetics professional for uncertain variants.
Warning And Precautions
- Avoid Galafold if you have a migalastat allergy.
- Inform your doctor about any history of kidney problems, and disclose pregnancy or breastfeeding status.
- Participation in a registry may occur for pregnant or breastfeeding individuals to monitor migalastat effects on the baby.
- Galafold isn’t suitable for those under 18.
- In case of an overdose, promptly contact your doctor.
Side effects Of Galafold
Galafold is generally well-tolerated, with the most common side effects being headache, nausea, and diarrhea. Other possible side effects include:
- Dizziness
- Fatigue
- Constipation
- Vomiting
- Abdominal pain
- Rash
Drug Interaction
A thorough understanding of potential drug interactions is essential for a holistic treatment strategy. Healthcare providers must be informed about any medications that could interact with Galafold, ensuring both its effectiveness and safety. Therefore, it is mandatory to inform your doctor about all current medications, including over-the-counter and prescription medicines, before initiating any treatment.
Conclusion
Galafold is a new medication that offers hope to patients with Fabry disease. It is the first oral medication to be approved for the treatment of this condition and has been shown to be effective in slowing the progression of the disease and improving the quality of life for patients. If you or someone you know has Fabry disease, talk to your doctor about Galafold. It may be the right treatment for you.
FAQs
Is Galafold suitable for everyone with Fabry disease?
Galafold is specifically indicated for adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant. Galafold is not approved for use in individuals younger than 18 years old.
What should I do if I miss a dose of Galafold?
If a dose is missed entirely, take it within 12 hours of the scheduled time. Otherwise, resume at the next planned dosing day.
How to buy Galafold online?
Purchasing Galafold has become more accessible online, particularly for individuals in the US and Europe. For those interested in acquiring this medication, consider contacting the SANSFRO team or other reputable organizations with expertise in importing medicines from these regions.
What is Galafold price in India?
As for the Galafold price, it varies based on specific product details and other influencing factors. For precise and up-to-date pricing information, we recommend reaching out to our dedicated Patient Support Team at (+91) 93157 05373 or sending an email to help@sansfro.com. Our team is equipped to provide accurate and current cost details for Galafold in the Indian market.
Reference
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