Cerdelga

Yes, the FDA approved Cerdelga (eliglustat) in August 2014. It's a medication that's taken by mouth and is approved for the long-term treatment of Gaucher disease type 1 (GD1) in certain adults.

The manufacturer of the brand-name medication Cerdelga is Genzyme Corporation.

Cerdelga (eliglustat) and Cerezyme (imiglucerase) are both first-choice treatment options for people with Gaucher disease type 1 (GD1), a genetic condition that causes a buildup of a fatty substance called GL-1 in the body. Cerdelga is a capsule that's taken by mouth every day. It helps lower the amount of GL-1 that your body makes so that Gl-1 can't build up. On the other hand, Cerezyme is given as an infusion into your veins (intravenously or IV) roughly every 2 weeks, typically at a treatment center. It's considered an enzyme replacement therapy (ERT); it replaces the missing enzyme (protein) in your body that helps break down GL-1 appropriately. Talk to your provider to see which of these two treatment options is right for you.

Cerdelga (eliglustat) and miglustat (Zavesca) work similarly to treat adults with Gaucher disease type 1 (GD1). They both block the body from making a fatty substance to help lessen toxic buildup in the organs. But Cerdelga (eliglustat) is considered a first-choice treatment option for GD1, whereas miglustat (Zavesca) isn't. The two medications both come as capsules that are taken by mouth, but miglustat (Zavesca) is typically taken more often during the day than Cerdelga (eliglustat). Cerdelga (eliglustat) appears to be less likely to cause side effects than miglustat (Zavesca). Speak with your provider to discuss which medication best fits your needs.

Your genetic makeup determines how well Cerdelga (eliglustat) gets broken down (metabolized) in your body. In people who can't metabolize Cerdelga (eliglustat) well, the medication can build up in your body and raise your risk for side effects, especially serious heart rhythm problems. Because of this, you might need to take Cerdelga (eliglustat) less often to prevent these side effects from happening. Before you start this medication, you must do genetic testing so your provider can find the right dose for you. Discuss with your provider about what your genetic test results mean for you.

It's not clear whether Cerdelga (eliglustat) is safe to take during pregnancy. There's not enough information about how the medication can affect you or your unborn baby if you take it while pregnant. Animal studies suggest that Cerdelga (eliglustat) can lead to birth defects. Keep in mind that there can be serious complications, such as a higher risk for miscarriage (pregnancy loss), if GD1 symptoms aren't controlled before or during pregnancy. Pregnancy can also worsen GD1 symptoms. Talk with your provider about the risks and benefits of taking Cerdelga (eliglustat) if you're pregnant or if you think you might become pregnant.

It's not known whether Cerdelga (eliglustat) can be found in breast milk, affect your milk supply, or harm your breastfed baby. But in animal studies, it was found in the milk of lactating rats, so it's possible that the medication can be passed along in human milk. Discuss with your provider about the risks and benefits of taking Cerdelga (eliglustat) if you’re breastfeeding.