Galafold

About Galafold 

• Galafold 123mg capsules, an oral pharmacological chaperone which contains the active ingredient called Migalastat Hydrochloride. 
• It is used to treat Fabry disease in adults with confirmed Galactosidase Alpha Gene (GLA) variant based on in vitro assay data. 
• In these individuals, Galafold functions by stabilizing the body’s own malfunctioning enzyme, enabling it to effectively reduce the buildup of disease-related substances.
• This approval is granted through an accelerated process, primarily due to the reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. 
• However, ongoing approval for this specific use may depend on further confirmation and a detailed account of the clinical benefits observed in subsequent confirmatory trials.
• Fabry disease is a rare genetic disorder that causes the buildup of a fatty substance called globotriaosylceramide (GL-3) in the body’s tissues and organs. This can lead to a variety of symptoms, including pain, fatigue, kidney disease, and heart disease.

Strength: 

Galafold capsules are delivered in a strength of 123mg respectively, for oral consumption.

Recommended Dosage:

Take Galafold on an empty stomach, which means you should avoid consuming any food for at least 2 hours before and 2 hours after taking Galafold. This creates a minimum 4-hour fasting period. However, you should feel free to drink clear liquids during this 4-hour fasting period. Additionally, it’s essential not to take Galafold on two consecutive days. Swallow capsules whole. Do not cut, crush, or chew

• The recommended dosage regimen for Galafold is 123mg to be taken orally once every other day at the same time of day. 
• Patients may need to adjust the dosage of medication according to their response and side effects. The recommended dosage of Galafold for your individual needs should be discussed with a healthcare professional.

Important:

Make sure not to miss any Galafold doses. If you do miss one, take it as soon as possible, but if your next dose is near, skip the missed dose and stick to your regular schedule; never double up. Keep your doctor informed about all your medications, including over-the-counter drugs, herbs, and vitamins, as some may interact with Galafold, potentially causing severe side effects. 

If you have questions or concerns about this medication, discuss them with your doctor. Following these instructions diligently is essential to maximize the medicine’s benefits and minimize the risk of side effects.

Warnings & Precautions

• Galafold can trigger a severe allergic reaction known as hypersensitivity reactions. Patients must undergo close monitoring for anaphylactic signs and symptoms during and after each infusion and discontinuation is needed if such reactions occur. 
• You should not consume this medicine if you are allergic to Galafold.
• Before starting to consume the capsule, inform your doctor if you have a history of kidney problems.
• Kidney function should be monitored in patients using Galafold, especially in patients with pre-existing kidney disease.
• Galafold has not been evaluated in individuals with Fabry disease whose estimated glomerular filtration rate (eGFR) is less than 30 mL/min/1.73 m2. Therefore, it is not advisable to use Galafold in patients with severe renal impairment or those in the advanced stage of renal disease necessitating dialysis. 
• No dosage modification is needed for individuals with mild to moderate renal impairment.
• Galafold showed no fetal developmental harm in pregnant rats and rabbits at doses up to 26 and 54 times the recommended human dose (based on (AUC) Area under the ROC Curve) and it had no adverse effects on postnatal development in rats when administered at doses up to 16 times the recommended human dose (based on AUC) during organogenesis and lactation.
• Inform your doctor if you are pregnant or breastfeeding, if you fall into either of these categories, your information may be recorded on a registry to monitor the effects of Galafold on the baby.
• Limited clinical data are available regarding Galafold use during pregnancy and its potential risks to fetal development. Its presence in human milk is unknown. Mothers should consider the benefits of breastfeeding alongside potential risks to the breastfed child and their clinical need for Galafold.
• It is unknown whether Galafold is safe to use in liver patients. So before starting the treatment inform your doctor if you have any pre-existing liver condition.  
• Galafold should be used with caution in patients taking other medications that may interact with the current medicine.

Common Galafold Side Effects:

• Headache
• Pyrexia 
• Nasopharyngitis
• Cough
• Vomiting
• Rash
• Nausea
• Urinary tract infection
• Fever
• Cold symptoms (e.g., stuffy or runny nose, sneezing)

Use in Specific Population

• Galafold is not approved for use in individuals under the age of 18. So, it is advised to mention your children’s age before starting the treatment. 
• The clinical trials for Galafold did not encompass an adequate number of individuals aged 65 and above to establish whether their response to the medication differs from that of younger patients.

Storage and Handling

Before handling Galafold capsules, ensure your hands are clean by washing them with soap and water. Avoid touching the capsules with wet hands. If you accidentally touch a capsule with wet hands, make sure your hands are completely dry before handling the capsule again. 

• Store Galafold capsules at room temperature of 20°C to 25°C (68°F to 77°F).
• Protect Galafold from light and moisture.
• Do not store Galafold in the bathroom or in other areas where it may be exposed to heat or humidity.
• Keep Galafold out of the reach of children and pets.