"The U.S. Food and Drug Administration yesterday approved Ruconest, the first recombinant C1-Esterase Inhibitor product for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).
(taliglucerase alfa) Injection, for Intravenous Use
Taliglucerase alfa, a hydrolytic lysosomal glucocerebroside-specific enzyme for intravenous infusion, is a recombinant active form of the lysosomal enzyme, β-glucocerebrosidase, which is expressed in genetically modified carrot plant root cells cultured in a disposable bioreactor system (ProCellEx®). β-Glucocerebrosidase (β-D-glucosyl-Nacylsphingosine glucohydrolase, E.C. 18.104.22.168) is a lysosomal glycoprotein enzyme that catalyzes the hydrolysis of the glycolipid glucocerebroside to glucose and ceramide.
ELELYSO is produced by recombinant DNA technology using plant cell culture (carrot). Purified taliglucerase alfa is a monomeric glycoprotein containing 4 N-linked glycosylation sites (Mr = 60,800). Taliglucerase alfa differs from native human glucocerebrosidase by two amino acids at the N terminal and up to 7 amino acids at the C terminal. Taliglucerase alfa is a glycosylated protein with oligosaccharide chains at the glycosylation sites having terminal mannose sugars. These mannose-terminated oligosaccharide chains of taliglucerase alfa are specifically recognized by endocytic carbohydrate receptors on macrophages, the cells that accumulate lipid in Gaucher disease.
ELELYSO is supplied as a sterile, non-pyrogenic, lyophilized product. The quantitative composition of each 200 unit vial is D-mannitol (206.7 mg), polysorbate 80 (0.56 mg), sodium citrate (30.4 mg), and taliglucerase alfa (212 units). Citric acid may be added to adjust the pH at the time of manufacture.
A Unit is the amount of enzyme that catalyzes the hydrolysis of 1 micromole of the synthetic substrate paranitrophenyl-β-D-glucopyranoside (pNP-Glc) per minute at 37 °C. After reconstitution with Sterile Water for Injection, taliglucerase alfa concentration is 40 units/mL [see DOSAGE AND ADMINISTRATION]. Reconstituted solutions have a pH of approximately 6.0.
What are the possible side effects of taliglucerase alfa (Elelyso)?
Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; blue lips or fingernails; feeling like you might pass out; swelling of your face, lips, tongue, or throat.
Some people receiving a taliglucerase alfa injection have had a reaction to the infusion (when the medicine is injected into the vein). Most infusion reactions have been mild. However, tell your caregiver right away if you feel dizzy, nauseated, anxious, light-headed, weak or tired, or have a sudden headache, red skin rash, fast or uneven heartbeats, chest...
Last reviewed on RxList: 9/5/2014
This monograph has been modified to include the generic and brand name in many instances.
Additional Elelyso Information
Report Problems to the Food and Drug Administration
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit the FDA MedWatch website or call 1-800-FDA-1088.
Find out what women really need.