Cerdelga
(eliglustat)
Get free couponsMiglustat
(Zavesca)
Get free couponsSummary for Eliglustat
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.
Indications for Eliglustat
- Gaucher disease type 1 (GD1) in certain adults
Pros and Cons for 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
Common Side Effects for Eliglustat
- Fatigue (14%)
- Headache (13%)
- Nausea (12%)
- Diarrhea (12%)
- Back pain (12%)
- Pain in arm or leg (11%)
- Upper stomach pain (10%)
Warnings for Eliglustat
- Heart rhythm problems
Summary for Miglustat
Miglustat (Zavesca) is an oral medication used to treat adults with mild to moderate type 1 Gaucher disease. It’s only used in people who can’t receive enzyme replacement therapy (ERT). Miglustat (Zavesca) is available as a generic medication so it might be less expensive than other branded medications approved for this condition.
Indications for Miglustat
- Mild to moderate type 1 Gaucher disease in adults
Pros and Cons for Miglustat
Pros
- Available as a lower-cost generic medication
- More convenient than frequent trips to the infusion center for ERT
- Is available as an oral medication, so it doesn’t require an IV or port compared to ERT
- Doesn't require genetic testing before starting like Cerdelga (eliglustat)
Cons
- Diarrhea and tremors are very common side effects
- Is taken three times a day in most adults
- Not for use in children or teenagers with type 1 Gaucher disease
- Can only be used if you can’t receive ERT
Common Side Effects for Miglustat
- Diarrhea (89-100%)
- Weight loss (39-67%)
- Stomach pain (18-67%)
- Passing gas (29-50%)
- Tremor (11-30%)
- Headache (21-22%)
- Nausea (8-22%)
- Weakness (17%)
- Vision problems (<17%)
- Vomiting (4-11%)
- Leg cramps (4-11%)
Warnings for Miglustat
- Nerve problems (peripheral neuropathy)
- Tremors
- Diarrhea and weight loss
- Lowering of platelet count
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