GoodRx Quarterly Report: Q1 2019
In an effort to continue to shed light on drug prices and increase transparency, we are excited to present the first ever GoodRx Quarterly Report—an
Cerdelga
Used for Inherited Metabolic Diseases, Lysosomal Storage Disease
Used for Inherited Metabolic Diseases, Lysosomal Storage Disease
Cerdelga (eliglustat) is a medication that belongs to a drug class known as glucosylceramide synthase inhibitors. This medication is FDA-approved as long-term treatment for certain adults with a rare genetic condition called Gaucher disease type 1 (GD1). Cerdelga (eliglustat) is a capsule that's taken by mouth. Side effects can include fatigue, headache, and nausea.
What is Cerdelga (eliglustat)?
What is Cerdelga (eliglustat) used for?
- Gaucher disease type 1 (GD1) in certain adults
How Cerdelga (eliglustat) works
People with Gaucher disease type 1 (GD1) have higher levels of a fatty substance called glucosylceramide (GL-1). As a result, GL-1 builds up in certain organs, like your spleen, liver, and bones, which can lead to serious symptoms and complications of GD1.
Cerdelga (eliglustat) is a glucosylceramide synthase inhibitor. It blocks your body from making GL-1. This lowers the amount of GL-1 in the body, which helps lower GL-1 buildup in your spleen, liver, and other organs.
Drug facts
| Common Brands | Cerdelga |
|---|---|
| Drug Class | Glucosylceramide synthase inhibitor |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
Side effects of Cerdelga (eliglustat)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Fatigue (14%)
- Headache (13%)
- Nausea (12%)
- Diarrhea (12%)
- Back pain (12%)
- Pain in arm or leg (11%)
- Upper stomach pain (10%)
Less Common Side Effects
- Dizziness
- Weakness
- Cough
- Indigestion or acid reflux
- Constipation
- Pounding heart (palpitations)
- Rash
Cerdelga (eliglustat) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Abnormal heart rhythm: abnormally fast heart rate, racing or pounding heart, fainting, dizziness
The following Cerdelga (eliglustat) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Heart rhythm changes—fast or irregular heartbeat, dizziness, feeling faint or lightheaded, chest pain, trouble breathing
Side effects that usually do not require medical attention (report to your care team if they continue or are bothersome):
Pros and cons of Cerdelga (eliglustat)
Pros
- First-choice treatment option in most adults with GD1
- Can take with or without food
- Taken by mouth, so it doesn’t require an IV or port like enzyme replacement therapy (ERT)
- More convenient than ERT since you don't need frequent trips to the infusion center
Cons
- Requires genetic testing before you start treatment
- Has many drug interactions (such as with certain antidepressants, antibiotics, and anti-seizure medications)
- Might not be able to use if you have certain liver, kidney, or heart problems because of the risk for serious side effects
- Not for use in children because it hasn’t been studied
Pharmacist tips for Cerdelga (eliglustat)
- Take Cerdelga (eliglustat) with or without food.
- Swallow Cerdelga (eliglustat) capsules whole, preferably with water. Don't open, crush, or dissolve the capsules because it might affect how the medication works. Let your provider know if you have trouble swallowing pills to see what other options you have.
- Avoid grapefruit and grapefruit juice while you're taking Cerdelga (eliglustat). Grapefruit can raise the amount of medication that your body absorbs, which means that your risk for side effects is greater.
- Tell your providers and pharmacists about all the medications and supplements you take so they can check for drug interactions with Cerdelga (eliglustat). These interactions can either make Cerdelga (eliglustat) work less well or raise your risk for side effects (e.g., heart rhythm problems). You might need some of your doses adjusted or stop some medications altogether to keep you safe.
- Tell your provider if you have heart, kidney, or liver problems. These health conditions might worsen or raise your risk for side effects. In some cases, your provider might need to change your Cerdelga (eliglustat) dose or stop the medication altogether.
Risks and warnings for Cerdelga (eliglustat)
Cerdelga (eliglustat) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Heart rhythm problems
- Risk factors: History of heart problems | Taking certain antiarrhythmics (medications for an irregular heartbeat)
Extremely high levels of Cerdelga (eliglustat) in the body can affect the way the heart beats and raise the risk for heart rhythm problems (arrhythmias). These heart rhythm problems can be serious and potentially life-threatening.
The levels of Cerdelga (eliglustat) you have in your body depend on how well your body can break down the medication. And how well your body breaks down the medication can depend on your genetic makeup and any medications you take that can interact with Cerdelga (eliglustat).
Tell your provider and pharmacist about your full medical history and all the medications you take. They can check your risk for heart rhythm problems with Cerdelga (eliglustat) treatment. You might need dose adjustments or an alternative treatment option. Avoid Cerdelga (eliglustat) if you have heart problems or if you take certain medications to control your heart rate, like amiodarone (Pacerone) and sotalol (Betapace).
Seek medical help right away if you experience an abnormally fast heartbeat, fainting, or dizziness while you're taking Cerdelga (eliglustat).
Cerdelga (eliglustat) dosage
Typical dosage for Cerdelga (eliglustat)
In general, the typical dose is 84 mg by mouth once or twice daily, with or without food.
Dosing is based on your genetic makeup and how well your body can break down Cerdelga (eliglustat). Because of this, you'll need to do genetic testing before you start this medication so your provider can prescribe the appropriate dose for you.
The dose will also depend on whether you have liver problems or whether you're taking other medications that can interact with Cerdelga (eliglustat).
Cerdelga (eliglustat) contraindications
Contraindications are specific health conditions or situations in which a person should not take a medication due to safety concerns. If you have any of the following conditions or if any of the following apply to you, let your healthcare provider know because Cerdelga (eliglustat) will not be safe for you to take.
- Taking certain medications that interact with Cerdelga (eliglustat), depending on your genetic makeup
- Certain liver problems, depending on your genetic makeup
What are alternatives to Cerdelga (eliglustat)?
There are a number of medications that your doctor can prescribe in place of Cerdelga (eliglustat). Compare a few possible alternatives below.
News about Cerdelga (eliglustat)
