Eliglustat is a prescription medication marketed under the name Cerdelga. It is intended for the treatment of a very race condition called type 1 Gaucher disease. It works as a substrate reduction therapy that functions by reducing the generation and accumulation of a protein that causes a very debilitating condition in the patient. The genetic condition known as Gaucher disease is characterized by the lack of an enzyme that breaks down lipids within the body and results in an unhealthy buildup that gradually harms the internal organs and causes terrible symptoms including hemorrhaging, anemia, joint pain, and weak bones that can break easily. This is also a degenerative disease that gets worse with time and needs reliable long-term treatment that Eliglustat provides.
The purpose of taking Eliglustat is to improve the condition of internal organs, skeletal structure, and blood cells for patients with Gaucher disease. It is only prescribed for patients with a very specific type of the rare disease which is determined by the metabolic rate of the liver enzyme 2D6 which determines the rate at which the body can break down drugs. While Eliglustat can be an effective treatment that can prevent injury or the need for more intense or invasive therapies, it is important to note that it is not a cure for the condition. While it may be used for purposes other than that prescribed, it has been designated as an orphan drug as a result of the rarity of the condition it was created to treat.
Virtually all medication can have unintended side effects on the patient, and Eliglustat is no exception. Although these side effects may not occur, all patients who take this drug should be aware that symptoms are possible prior to starting an Eliglustat regimen. If the following serious side effects occur, it is recommended that medical attention is sought out as soon as possible.
Some of the less common but serious side effects of Eliglustat include a racing heartbeat, unusually strong pulse, or other cardiovascular irregularities. Some patients have reported dizziness or fainting spells when using the medication. Additionally, Eliglustat has a certain danger of overdose which can be indicated by a number of symptoms. If a patient has any of these symptoms, immediate emergency medical care may be necessary.
Symptoms of an overdose include:
However, not all side effects of Eliglustat will necessarily require medical attention. Some side effects are common when first taking the medication, but subside as the body adjusts to having Eliglustat in the system. There may be some ways to mitigate these symptoms with the aid of a physician, but if they last longer than a few weeks or are more serious than normal, they may indicate that there is a deeper medical problem.
The most common of the temporary side effects can be intense and painful at first, including stomach problems, feeling bloated, experiencing digestive problems or gastrointestinal distress like diarrhea. This can often be accompanied by feeling gassy or excessive bleaching. Other common symptoms affect the musculoskeletal condition of the patient, resulting in back or muscle pain, pain in the joints or limbs, a feeling of weakness, or even difficulty moving at all. Other patients commonly report headaches and flu-like symptoms including coughing or a sore throat. Less common temporary side effects include a rash on the skin or difficulty moving the bowels. Since Eliglustat is a rarely prescribed medication for a rarely experienced disorder, side effects other than the ones listed may occur. The prescribing physician should be promptly informed of all symptoms experienced while taking this medication.
Eliglustat is a potent prescription medication that should only be taken as directed by the prescribing doctor. It should not be taken more often or for a longer duration than is indicated. While it is intended for long-term treatment, it may not be a perpetual answer to the ravages of this disorder. Using Eliglustat in any way other than as directed may result in an increased chance of side effects or overdose. When prescribed this medication, the directions included by the doctor should be read carefully and followed closely. The following information represents the guidelines from the manufacturer for an average patient but may be altered based on the specifics of a patient's condition.
When taking Eliglustat in capsule form it is recommended that it be swallowed whole with water to wash it down. It should not be chewed or broken open to be mixed with food. However, it can be taken at the same time as eating a meal if food helps the patient tolerate the dose. It also should not be taken within 24 hours of having taken the following medications:
Typical adult doses of Eliglustat usually follow the guidelines of the following doses:
CYP450 2D6 Extensive metabolizers (EMs) and Intermediate Metabolizers (IMs): 84 mg twice a day
CYP450 2D6 Poor Metabolizers (PMs): 84 mg once per day
EMs and IMs taking strong or moderate CYP450 2D6 inhibitors: 84 mg orally once per day
EMs taking strong or moderate CYP450 3A inhibitors: 84 mg orally once per day
When taking the medication in its capsule form for the treatment of type 1 Gaucher disease the initial dose will need to be determined by the results of a genotype blood test that will be administered by the attending physician. In the rare instance when it is prescribed to children the dose will need to be determined by a pediatric specialism based on the severity of the condition, the general health of the child, and body weight. If for any reason this designated dose is missed the dose should be skipped until the next dose on the schedule. This medicine comes with a risk of overdose so double dosing or taking doses too close to one another is potentially dangerous.
As with most medications, Eliglustat has a certain risk of interaction with other medications. Some of these medications should not be used at the same time as Eliglustat whatsoever, while others may be essential enough that the risk may be necessary. If there is a concern about these side effects your doctor may be able to make adjustments to a prescription including taking the medication at different times or in different amounts to reduce the instance of these interactions. Use of Eliglustat with any of the following medications is not recommended, and use of any of them may disqualify a patient from being able to take this medication.
Some other medications may not be usually recommended but may be permitted under certain circumstances. Proceed with care if using these medications at the same time as Eliglustat:
In addition to drug interactions, Eliglustat can have adverse reactions when used while consuming certain food, drinks, vitamins, supplements or other products consumed by the patient. While not all of these interactions are known, if there is a strange reaction to any of these while using the medication let your doctor know promptly. One of the most commonly known of these interactions is to grapefruit or grapefruit juice and this food should be avoided.
Certain medical conditions also have an interaction warning with Eliglustat and using the drug may make these conditions worse. Some of the conditions will also affect the medication or keep it from working as intended. If there is a history of any of these conditions or if they are currently being suffered, then this may affect the doctor's decision whether or not to prescribe this medicine.
Eliglustat is prescribed for a chronic rather than an acute condition and therefore is generally prescribed for long-term use. It is very important that during this interval that the patient attend regular visits for observation so that the prescribing physician can determine how well the prescription is working and if there are any problems developing as a result of using the medication. Eliglustat has been known to cause heart rhythm problems so the patient should inform a doctor of any unusual changes in heartbeat. If this is accompanied by dizziness or faintness this can be indicative of a serious health condition. Before using this medication the doctor should be made aware of any family history of heart problems. Prior to taking this medication, the prescribing physician should have a list of all prescription and non-prescription medication that is being taken as well as vitamins and supplements. These should not be taken unless discussed with the doctor who is prescribing Eliglustat.
Guidelines for storing this medication should be strictly observed as anyone taking this medication who does not have Gaucher disease can suffer very serious effects. This medication should be securely stored in its original container or another approved container that is clearly marked. Keep the pills at room temperature away from environmental sources that may damage them like heat, moisture, freezing temperatures, and direct sunlight. This medication should not be refrigerated or frozen and should be kept well out of the reach of children. If the prescription is expired or no longer needed, dispose of it in keeping with your local guidelines pertaining to the disposition of medical waste. If you have any questions about this process ask your doctor or pharmacist before simply throwing it away.
Patients who suffer from Type 1 Gaucher Disease are rare, but their suffering, when afflicted with this genetic disorder, can be profound. Despite the potential for side effects and poor interaction with other necessary medications, Eliglustat may represent the best chance for a patient to live with the debilitating symptoms of type 1 Gaucher disease. Under ideal conditions, it is effective in balancing the body's metabolic breakdown of the harmful protein that causes the fatty buildup causes the most damage to people in this condition but its effectiveness can be adversely affected by a number of other necessary treatments. The biggest hurdle faced with this drug often has more to do with the rarity of the condition that it is designed to treat. As such a rarely-prescribed treatment it has been classified as an orphan drug and therefore is often only sold at a very high price that many insurance companies may be reluctant to cover. However, this should not be a consideration when determining its value as a treatment for Gaucher disease.