Description:The two glucosylceramide synthase inhibitors on the market are eliglustat (Cerdelga) and miglustat (Zavesca). They are used to treat patients with type 1 Gaucher’s disease, a rare condition.
Serious Side Effects
Severe side effects of glucosylceramide synthase inhibitors include:
Such side effects can be severe or life-threatening.
Common Side Effects
Cerdelga may be associated with these common side effects: upper abdominal or stomach pain, sore throat, passing gas, pain in the arms or legs, muscle pain or stiffness, lack or loss of strength, indigestion, heartburn, full feeling, excess air or gas in the stomach or intestines, difficulty moving, diarrhea, cough, bloating, belching, back pain and sour stomach. Similar side effects occur with Zavesca, but with the added symptoms: disturbed color perception, unsteady walk, dizziness, double vision. If any side effects continue or worsen, contact your physician right away.
Mechanism of Action
A deficiency or absence of the enzyme b-glucocerebrosidase results in the abnormal accumulation of glucosylceramide. The clinical spectrum of this disease is wide, from patients that are asymptomatic and may have anemia or an enlarged spleen, to patients that have significant central nervous system problems such as: easy bruising or bleeding, anemia, enlarged liver or spleen, bone or joint pain, and weakened bones.
Both inhibitors on the market, eliglustat (Cerdelga) and miglustat (Zavesca) were developed for Gaucher’s disease and have had success in treating symptoms. These drugs were reported to have good oral availability, a high therapeutic index, and limited toxicity.
The drugs work by helping to relieve some of the blood, liver, spleen, and other organ problems that patients with type 1 Gaucher’s disease experience. Neither Cerdelga nor Zavesca are recommended for patients with liver impairment, heart disease, kidney problems or any other medication that interacts with Cerdelga or Zavesca.
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Contributors to this Article: Michael Sapko, MD, PhD; Deborah Kallick, PhD, Medicinal Chemistry