eliglustat (Cerdelga)

Indications

• long-term treatment of adults with Gaucher disease type 1

Dosage

• start 42 mg PO BID
• increase dose in 4 weeks
• usual dose 84 mg PO BID

Capsule

Adverse effects

• fatigue
• headache
• nausea
• diarrhea
• back pain
• pain in extremities
• upper abdominal pain

Mechanism of action

• slows production of fatty material spleen, liver, & bone marrow
• similar efficacy to enzyme replacement with imiglucerase

More general terms

• metabolic agent (metabolic modifier)
• aromatic compound
• ether
• amide
• alcohol
• pyrrolidine; tetrahydropyrrole

Database

• PubChem: http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?cid=23652731
• PubChem: http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?cid=52918379