Cerdelga Approved for Type 1 Gaucher Disease

Sanofi and the FDA announced in late August, the approval of a new drug used to treat type 1 Gaucher disease. Eliglustat, or Cerdelga, is approved for the long-term treatment of the condition. Patients with type 1 Gaucher disease have a certain drug-metabolizing enzyme genotype, and this drug is designed to partially inhibit the production of the enzyme that makes glucocerebroside.

Patients with type 1 Gaucher disease cannot adequately catabolize glucocerebroside, a fat molecule found in the cells of the brain, nervous tissue, and in other tissues throughout the body. Because the body cannot properly break down the fat, it leads to a buildup of fatty material in the bone marrow, liver, and spleen. This results in liver and spleen enlargement, as well as low platelet counts, anemia, and bone problems.

Clinicians must choose patients to treat based on their genotype for cytochrome P-450 isoenzyme 2D6. The medication is a substrate of CYP2D6. This drug is recommended for people who are: poor, intermediate, or extensive metabolizers of CYP2D6 substrates. It is not recommended for people who are ultra-rapid metabolizers, or for patients whose CYP2D6 genotype cannot be determined.

Patients who are either extensive or intermediate metabolizers should take 84 mg of the medication twice daily, by mouth. Patients who are poor metabolizers should take the 84 mg dose by mouth once a day. These results were determined by pharmacokinetic prediction rather than a clinical study.

Beyond being a substrate for CYP2D6, the medication is also a substrate of CYP3A. It is not recommended for patients who are CYP2D6 extensive or intermediate metabolizers who use a strong or moderate CYP2D6 inhibitor along with a strong or moderate CYP3A inhibitor. The medication should also not be used with patients who are considered either an intermediate or poor metabolizer, who are also using a CYP3A inhibitor.

The FDA required medication labeling provides information on how drugs will affect eliglustat, along with dose adjustment recommendations, and recommendations for clinicians. The use of other medications, or herbal supplements may increase the risk of side effects. St. John’s Wort, along with medications used to treat seizures, fungal infections, heart conditions, depression, tuberculosis, and hypertension could increase the risk for side effects, so patients should always tell their doctor or pharmacist if they are under any prescription or over the counter treatment for any of these conditions. Patients should never start a new medication, be it prescription or over the counter without first talking to their doctor and pharmacist.

The most common side effects reported with this medication are: headache, nausea, fatigue, back pain, diarrhea, and pain in the extremities and/or upper abdomen.

Cerdelga is available in capsules containing 84 mg of eliglustat – 100 mg of eliglustat hemitartrate. Capsules are packaged 14 per blister card, with one or four blister packs per carton. The medication should be stored at 20–25 °C (60-77 °F).