Overview:
Miglustat was first approved by the Food and Drug Administration (FDA) in the year 2000 to treat Gaucher’s disease. The drug has been approved in different countries like Canada, Japan, and the European Union. The drug is yet to be approved for the treatment of other conditions like Niemann-Pick disease.
How Does Miglustat Work?
Miglustat is an enzyme inhibitor that is used in the long-term treatment of certain genetic disorders like Gaucher’s disease. The drug works by inhibiting glucosylceramide synthase, which is primarily responsible for producing certain fatty substances called lipids. The deficiency of this enzyme could result in the accumulation of fatty substances in the body, like the liver, bone marrow, and spleen.
Uses:
Miglustat is used in the treatment of the following diseases
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Gaucher’s disease (building of fatty substances in various organs).
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Niemann-Pick disease type C (a progressive genetic disorder).
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Type 1b mucopolysaccharidosis (lysosomal storage disorder).
In the disease mentioned above, the drug acts by inhibiting the enzymes, thereby preventing the accumulation of fatty substances in various organs and improving the symptoms of the disorders.
Dosage:
The drug is available in the form of a capsule, which has to be consumed orally, with or without food. The dosage depends on the type of disorder being treated. In some cases, the dosage may be altered according to the patient’s response to the treatment. Following the dosage recommended by the healthcare professional is crucial for better results.
Warnings:
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Inform the doctor regarding any allergic reactions to the drug.
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In the case of planning to become pregnant, it is better to inform the healthcare professional as it could affect the child (fetus).
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The drug could affect the sperm, so men are recommended to use birth control measures during treatment and three months post-treatment to prevent unwanted complications.
For Patients
What Is Gaucher Disease?
Gaucher disease is the buildup of fatty substances in particular organs like the spleen and liver. As a result, the organs enlarge and can affect their function. Sometimes, these fatty substances could also be observed in bone tissues, thereby increasing bone fractures. The commonly associated symptoms are listed below.
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Bone pain.
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Fatigue.
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Delayed growth and puberty.
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Enlargement of the spleen.
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Low platelet counts.
There are three types of Gaucher disease, of which type 1 is the most common form seen in adults. Managing the symptoms can help to decrease the disease progression.
Learn More About Miglustat
When and How Often to Take Miglustat?
The dosage for Gaucher disease is 100 milligrams thrice a day. It is recommended to consume the drug along with food to reduce the risk of gastrointestinal problems.
How Effective Is Miglustat?
The drug is highly effective in reducing the symptoms of Gaucher disease. However, the drug helps to improve the symptoms rather than cure the disease.
Things to Inform The Doctor Before They Prescribe Miglustat:
Inform the doctor regarding any allergic reactions and previous medical history before they prescribe the drug to prevent unwanted drug interactions.
How to Take Miglustat?
The drug is to be consumed with meals or after meals. Always consume the recommended dose as prescribed by the doctor for better benefits.
Look Out for Side Effects:
Some of the side effects of miglustat are listed below. In case of any such side effects, inform the healthcare professional and take treatment for the symptoms at the earliest possible.
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Gas.
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Loss of appetite.
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Upset stomach.
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Vomiting.
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Constipation.
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Dry mouth.
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Weakness.
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Memory disorders.
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Irregular menstrual cycle.
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Poor gait (walking style).
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Frequent muscle cramps.
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Nausea.
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Dry mouth or xerostomia.
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Back pain.
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Nervousness.
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Fatigue or weakness.
In some cases, patients may experience shivering hands, changes in their vision, and easy bruising. However, these symptoms are uncommon.
Dietary Alterations:
The drug may cause diarrhea and weight loss, so it is important to change or plan the diet in a way that may improve the immune system and help fight diseases efficiently.
What Should Be Done When a Dose Is Missed?
In case of missing a dose, consume the dose at the earliest possible as it aids with treatment progression. Avoid double dosing as it could be harmful and cause serious side effects.
What Should Be Done to Treat Miglustat Overdose?
In case of a Miglustat overdose, inform the healthcare professional at the earliest possible for better assistance. Some of the symptoms of overdose include
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Fever.
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Sore throat.
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Chills.
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Signs of infection.
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Dizziness.
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Numbness in the hands and legs.
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Burning sensation.
How to Store Miglustat?
The drug should be stored at room temperature in a dry place away from sunlight. It is crucial to keep the medication in its original packing and avoid transferring it to other containers, as it could contaminate the drug. Keep it out of reach of children for accidental ingestion, and do not store it in damp areas. Moisture could interfere with the quality and effectiveness of the drug, so proper storing can help reap better benefits. Proper storage of any drug ensures environmental safety and better benefits to treat the condition appropriately.
How to Handle Miglustat?
Before handling Miglustat, taking a few precautions can ensure the safety and effectiveness of the drug.
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Wash hands thoroughly before and after handling the medication.
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Using gloves and a small instrument to pick the drug can be helpful and prevent cross-contamination of drugs.
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Avoid consuming more than the recommended dose, as it could cause complications.
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In case of any allergic reactions or side effects, inform the healthcare professional immediately.
How to Dispose of Miglustat?
Before consuming the drug, it is better to check for expiry and dispose of it according to the local regulations. Avoid flushing the tablets in the toilet and pouring them down the drain, as they may harm the environment.
Avoid Self-Medication:
Self-medication can be harmful if the patient is not properly aware of the condition. Strictly following the doses recommended by the doctor can benefit health and help lead a healthy life.
Tips to Stay On Track:
Always consume the drug at the same time every day as it helps to avoid skipping the drug accidentally. Set the alarm or reminder on the phone to aid with consuming the drug on time.
For Doctors
Indication:
Miglustat is used in specific genetic disorders that cause the accumulation of fatty substances in various parts of the body. Some examples of genetic disorders that can be treated with Miglustat include Gaucher disease and Niemann-Pick disease.
Pharmacology:
Taxonomy
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Kingdom -Organic compounds.
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Subkingdom - Organic oxygen compounds.
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Superclass - Organooxygen compounds.
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Class - Carbohydrates and conjugates.
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Subclass - Glycosylamines.
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Molecular Formula - C8H17NO6
Mechanism of Action:
The drug inhibits the enzyme glucosylceramide synthase, which is actively involved in producing glycosphingolipids. Inhibiting the enzyme helps the production of fatty substances that accumulate in various body parts.
The drug has been proven to reduce the levels of glycosphingolipids in different organs and improve the symptoms of the disorder.
Pharmacodynamics:
The pharmacodynamics of the drug depends on the type of disorder being treated. Generally, the drug works by producing specific lipids, which decreases the accumulation in different organs and improves the symptoms of genetic disorders. However, the drug dosage has to be altered according to the patient’s requirements.
Ingredients:
Active Ingredient:
Miglustat hydrochloride is the active ingredient of the drug, which inhibits glucosylceramide synthase, an enzyme. The drug is a crystalline powder that is soluble in water.
Inactive Ingredients:
The inactive ingredients of the drug include cellulose, which is a bulking agent and a binder. In addition, croscarmellose sodium is used for the disintegration of the tablet. Apart from the above-mentioned, magnesium stearate is used as a lubricant, and gelatin is a form of collagen used as the capsule shell. The white color of the drug is due to titanium oxide, a whitening agent.
Absorption:
The drug is rapidly absorbed and has a bioavailability of 97 percent. The time taken to achieve the plasma concentration is about two to four hours.
Distribution:
The drug is distributed via the bloodstream to specific body parts, where there is the accumulation of fatty deposits and reduces the build-up to lead a healthy life.
Metabolism:
The drug is metabolized in the liver, where it undergoes conversion and is transported to other parts of the body. Circulation of the drug to different body parts helps to remove the fatty deposits.
Elimination:
The drug is eliminated by the kidneys after complete metabolism in the liver and distribution to different parts to treat the condition.
Toxicity:
The drug causes various toxicity, some of which are mentioned below
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Gastrointestinal toxicity like nausea, diarrhea, and abdominal pain.
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Electrolyte imbalance with severe dehydration.
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Peripheral neuropathy (damage to the nerves surrounding the brain and spinal cord).
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Tremors and ataxia.
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Hepatotoxicity (liver injury).
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Teratogenicity (birth defects in children).
Warning and Precaution:
The drug is not safe to be used in pregnant women as it could cause birth defects. Similarly, more caution is required before administering the drug in hepatic and renal impaired patients, as it affects the drug's efficacy.
Administration of the Drug:
The drug is administered orally along with food. It is available in the form of an oral tablet or capsule, which is readily soluble in water. Consuming the drug on a timely basis can help to decrease the symptoms and treat the disease effectively.
Considerations for Administration:
Contraindications:
The drug is contraindicated in patients with hypersensitivity. Also, patients with several renal impairments are not recommended for Miglustat. The drug is teratogenic and cannot be prescribed to pregnant mothers. It is important to inform the doctor regarding any previous history of allergies to help them to prescribe an alternative medication.
Clinical Studies for Miglustat:
Several clinical studies conducted on Miglustat have shown that it is the primary drug for the treatment of Gaucher and Niemann-pick disease. The clinical trials evaluated the drug's safety and efficacy and proved significant improvement in neurological symptoms. However, the most common side effects are diarrhea and abdominal pain.
Drug Interactions:
Miglustat interacts with other drugs and affects their efficacy. Some examples include CYP2D6 inhibitors like Paroxetine, which increases the plasma concentration of Miglustat, thereby resulting in adverse effects.
Other Specifications
Miglustat in Pregnant Women:
There is limited information regarding the use of drugs in pregnant women as it is generally not advised during pregnancy. Due to potential risks, women planning to become pregnant should inform the healthcare professional priorly to help them plan the treatment accordingly. If the administration of the drug is strongly recommended during pregnancy, then monitoring the fetus is the safer option to prevent developmental abnormalities.
Miglustat in Lactating Women:
The excretion of Miglustat in human milk is yet to be known. Hence it is not recommended for breastfeeding mothers, as it could potentially affect the infant. So before consuming the drug, it is better to measure the benefits and risks.
Miglustat in Pediatric Patients:
The drug has been approved for treatment in pediatric patients with progressive neurological manifestations. However, limited studies are available on the safety and efficacy of the population. The dosing for children is based on the weight of the body and the severity of the condition. Close monitoring of adverse effects is strongly recommended.
Miglustat in Geriatric Patients:
The drug action in elderly patients does not vary much from younger individuals. So no dosage treatment adjustment is required unless there are pre-existing medical conditions. Geriatric patients are susceptible to dizziness, tremor, and confusion. So it is crucial to closely monitor such patients. In addition, any kidney or liver disease can influence the absorption, metabolism, and elimination of the drug. So dosage adjustments are made in such specific patients for the success of the treatment.
Miglustat in Renal Impairment Patients:
The drug is eliminated from the kidneys, so kidney impairment may affect the elimination process, resulting in surplus drugs circulating in the bloodstream. Therefore, the drug should be used cautiously in renal-impaired patients. If deemed necessary in patients with moderate renal impairment, the recommended starting dose of 100 milligrams is administered once daily. Close monitoring and renal function tests are done to check for adverse effects.
Miglustat in Hepatic Impairment Patients:
The drug is primarily metabolized in the liver, so the pharmacokinetics may be affected by hepatic impairment. With hepatic impairment, there is decreased ability to metabolize the drug, resulting in the highest systemic exposure. Hence dosage adjustments are strongly recommended in hepatic-impaired patients before drug administration.
