Medications for Gaucher Disease
These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Gaucher Disease.
Found 5 Approved Drugs for Gaucher Disease
Cerezyme
Generic Name
Imiglucerase
Cerezyme
Generic Name
Imiglucerase
Form: Injection
Method of administration: Intravenous
FDA approval date: May 23, 1994
Classification: Hydrolytic Lysosomal Glucocerebroside-specific Enzyme
Cerezyme is indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions: anemia thrombocytopenia bone disease hepatomegaly or splenomegaly Cerezyme is a hydrolytic lysosomal glucocerebrosidase-specific enzyme indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions: anemia, thrombocytopenia, bone disease, hepatomegaly or splenomegaly. ( 1 )
VPRIV
Generic Name
Velaglucerase
VPRIV
Generic Name
Velaglucerase
Form: Injection
Method of administration: Intravenous
FDA approval date: February 26, 2010
Classification: Hydrolytic Lysosomal Glucocerebroside-specific Enzyme
VPRIV is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. ( 1 )
Cerdelga
Generic Name
Eliglustat
Cerdelga
Generic Name
Eliglustat
Form: Capsule
Method of administration: Oral
FDA approval date: September 03, 2014
Classification: Glucosylceramide Synthase Inhibitor
CERDELGA is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test [see Dosage and Administration.
Elelyso
Generic Name
Taliglucerase
Elelyso
Generic Name
Taliglucerase
Form: Injection
Method of administration: Intravenous
FDA approval date: May 01, 2012
Classification: Hydrolytic Lysosomal Glucocerebroside-specific Enzyme
ELELYSO is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease. ELELYSO is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease ( 1 ).
Miglustat
Brand Names
Yargesa, Zavesca, Opfolda
Miglustat
Brand Names
Yargesa, Zavesca, Opfolda
Form: Capsule
Method of administration: Oral
FDA approval date: July 31, 2003
Classification: Glucosylceramide Synthase Inhibitor
OPFOLDA is indicated, in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). OPFOLDA is an enzyme stabilizer indicated, in combination with Pombiliti, a hydrolytic lysosomal glycogen-specific enzyme, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). ( 1 )
Showing 1-5 of 5
Not sure about your diagnosis?
Check Your Symptoms
Tired of the same old research?
Check Latest Advances