Last reviewed 2026-04-20 · How we verify

Rhgaa (ALGLUCOSIDASE ALFA)

Rhgaa works by replacing the deficient enzyme in patients with Pompe disease.

Rhgaa (ALGLUCOSIDASE ALFA) is a hydrolytic lysosomal glycogen-specific enzyme developed by Genzyme, currently owned by the same company. It is a small molecule modality approved by the FDA in 2006 for the treatment of lysosomal alpha-1,4-glucosidase deficiency in infantile onset. The commercial status of Rhgaa is patented, and it is used to treat a specific genetic disorder. Key safety considerations include its short half-life of 2.3 hours. Rhgaa works by replacing the deficient enzyme in patients with Pompe disease.

At a glance

Mechanism of action

Pompe disease (acid maltase deficiency, glycogen storage disease type II, GSD II, glycogenosis type II) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA.Alglucosidase alfa provides an exogenous source of GAA. Binding to mannose-6-phosphate receptors on the cell surface has been shown to occur via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen.

Approved indications

• Lysosomal alpha-1,4-glucosidase deficiency - infantile onset

Boxed warnings

• WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE See full prescribing information for complete boxed warning. Hypersensitivity Reactions Including Anaphylaxis Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate LUMIZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue LUMIZYME and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 ) Immune-Mediated Reactions Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following LUMIZYME treatment. Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs while receiving LUMIZYME. ( 5.3 ) Risk of Acute Cardiorespiratory Failure Infantile-onset Pompe disease (IOPD) patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload and require additional monitoring. ( 5.4 ) Hypersensitivity Reactions Including Anaphylaxis Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate LUMIZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue LUMIZYME and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ]. Immune-Mediated Reactions Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following LUMIZYME treatment. Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs while receiving LUMIZYME [see Warnings and Precautions (5.3) ] . Risk of Acute Cardiorespiratory Failure Infantile-onset Pompe disease (IOPD) patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload and require additional monitoring [see Warnings and Precautions (5.4) ] .

Common side effects

• Anaphylaxis
• Hypersensitivity reactions
• Rash
• Pyrexia
• Urticaria
• Flushing
• Hypertension/Increased Blood Pressure
• Decreased Oxygen Saturation
• Cough
• Tachypnea
• Tachycardia
• Erythema

Key clinical trials

• PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (PHASE1)
• A Global Prospective Observational Registry of Patients With Pompe Disease
• A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa (PHASE2)
• Pompe Pregnancy Sub-Registry
• Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PHASE3)
• Expanded Access for ATB200/AT2221 for the Treatment of IOPD
• Drug-drug Interaction Study (PHASE2)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Rhgaa CI brief — competitive landscape report
• Rhgaa updates RSS · CI watch RSS
• Sanofi portfolio CI