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
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
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
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
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
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 )
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