Velaglucerase alfa, sold under the brand name Vpriv and manufactured by

Shire plc Shire plc was a UK-founded Jersey-registered specialty biopharmaceutical company. Originating in the United Kingdom with an operational base in the United States, its brands and products included Vyvanse, Lialda, and Adderall XR. Shire was ac ...

, is a hydrolytic

lysosomal A lysosome () is a membrane-bound organelle found in many animal Cell (biology), cells. They are spherical Vesicle (biology and chemistry), vesicles that contain Hydrolysis, hydrolytic enzymes that can break down many kinds of biomolecules. A ly ...

glucocerebroside Glucocerebroside (also called glucosylceramide) is any of the cerebrosides in which the monosaccharide head group is glucose. Clinical significance In Gaucher disease, the enzyme glucocerebrosidase is nonfunctional and cannot break down glucocer ...

-specific enzyme, which is a recombinant form of

glucocerebrosidase β-Glucocerebrosidase (also called acid β-glucosidase, D-glucosyl-N-acylsphingosine glucohydrolase, or GCase) is an enzyme with glucosylceramidase activity () that is needed to cleave, by hydrolysis, the beta-glycosidic linkage of the chemical g ...

indicated as a long-term enzyme replacement therapy for those with

Gaucher disease Gaucher's disease or Gaucher disease () (GD) is a genetic disorder in which glucocerebroside (a sphingolipid, also known as glucosylceramide) accumulates in cells and certain organs. The disorder is characterized by bruising, fatigue, anemia, low ...

Type 1. It has an identical amino acid sequence to the naturally occurring enzyme. It was approved for use by the U.S.

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...

(FDA) on February 26, 2010. The most common side effects include abdominal (belly) pain, headache, dizziness, bone pain, arthralgia (joint pain), back pain, infusion-related reactions, asthenia (weakness) or fatigue (tiredness), and pyrexia (fever) or increased body temperature. Velaglucerase alfa was approved for medical use in the European Union in August 2010. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

Medical uses

Velaglucerase alfa is indicated for long-term enzyme-replacement therapy (ERT) in people with type-1 Gaucher disease.

Competitive products

Imiglucerase Imiglucerase is a medication used in the treatment of Gaucher's disease. It is a recombinant DNA-produced analogue of the human enzyme β-glucocerebrosidase. Cerezyme is a freeze-dried medicine containing imiglucerase, manufactured by Genzyme C ...

Genzyme Genzyme was an American biotechnology company based in Cambridge, Massachusetts. Since its acquisition in 2011, Genzyme (also known as Genzyme Transgenics Corp or GTC Biotherapeutics) has been a fully owned subsidiary of Sanofi. In 2010, Genzyme ...

's version of recombinant glucocerebrosidase. It is marketed globally under the trade name of Cerezyme. In addition,

Protalix Protalix BioTherapeutics is an Israeli pharmaceutical company that manufactures a plant-based enzyme, taliglucerase alfa, which has received U.S. Food and Drug Administration approval for the treatment of Gaucher disease. Corporate history Prot ...

and

Pfizer Pfizer Inc. ( ) is an American multinational pharmaceutical and biotechnology corporation headquartered on 42nd Street in Manhattan, New York City. The company was established in 1849 in New York by two German entrepreneurs, Charles Pfizer ...

have received approval for marketing

taliglucerase alpha Taliglucerase alfa, sold under the brand name Elelyso among others, is a biopharmaceutical medication developed by Protalix and Pfizer. The drug, a recombinant glucocerebrosidase used to treat Gaucher's disease, is the first plant-made pharmace ...

in Israel, Brazil, Canada and the U.S. The companies are expected also to file for marketing authorization in Europe and elsewhere around the world.

References

External links

* Recombinant proteins Orphan drugs Takeda Pharmaceutical Company brands {{gastrointestinal-drug-stub