Alglucosidase alfa
Encyclopedia
Alglucosidase alfa is an enzyme replacement therapy
(ERT) orphan drug
for treatment of Pompe disease (Glycogen storage disease type II
), a rare
lysosomal storage disorder (LSD).
Chemically speaking, the drug is an analog
of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase
. It is the first drug available to treat this disease.
pharmaceutical company, Genzyme
, markets alglucosidase alfa as "Myozyme". In 2006, the U.S. Food and Drug Administration (FDA) approved Myozyme as a suitable ERT treatment for children.
Some health plans have refused to subsidize Myozyme for adult patients because it lacks approval for treatment in adults, as well as its high cost (US$300,000/yr for life).
, respiratory complications, infection
s and fever
. More serious reactions reported include heart and lung failure and allergic shock
. Myozyme boxes carry warnings regarding the possibility of life-threatening allergic response.
Enzyme replacement therapy
Enzyme replacement therapy is a medical treatment replacing an enzyme in patients in whom that particular enzyme is deficient or absent. Usually this is done by giving the patient an intravenous infusion containing the enzyme...
(ERT) orphan drug
Orphan drug
An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease...
for treatment of Pompe disease (Glycogen storage disease type II
Glycogen storage disease type II
Glycogen storage disease type II is an autosomal recessive metabolic disorder which damages muscle and nerve cells throughout the body. It is caused by an accumulation of glycogen in the lysosome due to deficiency of the lysosomal acid alpha-glucosidase enzyme...
), a rare
Rare disease
A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population.Most rare diseases are genetic, and thus are present throughout the person's entire life, even if symptoms do not immediately appear...
lysosomal storage disorder (LSD).
Chemically speaking, the drug is an analog
Analog (chemistry)
In chemistry, a structural analog , also known as chemical analog or simply analog, is a compound having a structure similar to that of another one, but differing from it in respect of a certain component. It can differ in one or more atoms, functional groups, or substructures, which are replaced...
of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase
Alpha-glucosidase
Alpha-glucosidase is a glucosidase acting upon 1,4-alpha bonds. This is in contrast to beta-glucosidase.The term maltase, which refers to an enzyme cleaving maltose, is nearly equivalent.Types include:* Acid alpha-glucosidase* neutral** GANAB** GANC...
. It is the first drug available to treat this disease.
Status
Orphan drugOrphan drug
An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease...
pharmaceutical company, Genzyme
Genzyme
Genzyme Corporation is a fully owned subsidiary of Sanofi-Aventis. Before its acquisition, Genzyme was an American biotechnology company based in Cambridge, Massachusetts. In 2010, Genzyme was the world’s third-largest biotechnology company, employing more than 11,000 people around the world...
, markets alglucosidase alfa as "Myozyme". In 2006, the U.S. Food and Drug Administration (FDA) approved Myozyme as a suitable ERT treatment for children.
Some health plans have refused to subsidize Myozyme for adult patients because it lacks approval for treatment in adults, as well as its high cost (US$300,000/yr for life).
Side effects
Common observed adverse reactions to alglucosidase alfa treatment are pneumoniaPneumonia
Pneumonia is an inflammatory condition of the lung—especially affecting the microscopic air sacs —associated with fever, chest symptoms, and a lack of air space on a chest X-ray. Pneumonia is typically caused by an infection but there are a number of other causes...
, respiratory complications, infection
Infection
An infection is the colonization of a host organism by parasite species. Infecting parasites seek to use the host's resources to reproduce, often resulting in disease...
s and fever
Fever
Fever is a common medical sign characterized by an elevation of temperature above the normal range of due to an increase in the body temperature regulatory set-point. This increase in set-point triggers increased muscle tone and shivering.As a person's temperature increases, there is, in...
. More serious reactions reported include heart and lung failure and allergic shock
Allergy
An Allergy is a hypersensitivity disorder of the immune system. Allergic reactions occur when a person's immune system reacts to normally harmless substances in the environment. A substance that causes a reaction is called an allergen. These reactions are acquired, predictable, and rapid...
. Myozyme boxes carry warnings regarding the possibility of life-threatening allergic response.
External links
- Myozyme (alglucosidase alfa), Genzyme official website
- MTAP (Myozyme Temporary Access Program), Genzyme official website