Alglucosidase alfa

Alglucosidase alfa Brand Names- Lumizyme | Myozyme

What is Alglucosidase alfa

Recombinant human alglucosidase alfa (rhGAA; Myozyme, Lumizyme) is an enzyme replacement therapy for endogenous acid alpha-glucosidase.

Myozyme and Lumizyme, produced by recombinant DNA technology in a Chinese hamster ovary cell line, are used in the treatment of Pompe disease (glycogen storage disease type II, GSD II).

Pompe disease, a rare disease affecting approximately 1:40,000 (an estimated 5,000—10,000 people worldwide), is caused by a genetic deficiency of the enzyme acid alpha-glucosidase, which is responsible for breaking down glycogen within lysosomes, resulting in intralysosomal accumulation of glycogen.

In infantile-onset Pompe disease, where the deficiency in acid alpha-glucosidase is absolute, accumulation of glycogen occurs primarily in the cardiac, hepatic, and skeletal muscles leading to cardiomyopathy, progressive muscle weakness, and impairment of respiratory function.

In late-onset Pompe disease, a less aggressive type with relative deficiency of acid alpha-glucosidase, accumulation of glycogen is limited primarily to the skeletal muscles, which leads to progressive muscle weakness and eventual confinement to a wheelchair and respiratory failure; severe cardiac involvement is rarely apparent in the late-onset form.

For both types of Pompe disease, respiratory failure is the usual cause of death; without treatment, the infantile-onset type is rapidly lethal by the age of 1 whereas death in patients with late-onset Pompe disease ranges from early childhood to late adulthood depending on the rate of disease progression. 

Lumizyme was approved by the FDA for patients 8 years and older with late (non-infantile) onset Pompe disease who do not have evidence of cardiac hypertrophy in May 2010; in August 2014, FDA approval was expanded to include all patients with Pompe disease.

Indications

  • acid alpha-glucosidase deficiency (Pompe disease)

Side Effects

  1. abdominal pain
  2. agitation
  3. anaphylactic shock
  4. anemia
  5. angioedema
  6. antibody formation
  7. apnea
  8. arthralgia
  9. arthropathy
  10. bradycardia
  11. bronchospasm
  12. candidiasis
  13. cardiac arrest
  14. chest pain (unspecified)
  15. chills
  16. conjunctivitis
  17. constipation
  18. cough
  19. cyanosis
  20. diaper dermatitis
  21. diarrhea
  22. dizziness
  23. dyspnea
  24. edema
  25. erythema
  26. fatigue
  27. fever
  28. flushing
  29. gastroesophageal reflux
  30. glomerulonephritis
  31. headache
  32. heart failure
  33. hyperhidrosis
  34. hypertension
  35. hypotension
  36. hypoxia
  37. infection
  38. infusion-related reactions
  39. irritability
  40. lacrimation
  41. livedo reticularis
  42. maculopapular rash
  43. malaise
  44. muscle cramps
  45. myalgia
  46. nausea
  47. nephrotic syndrome
  48. pallor
  49. paresthesias
  50. peripheral edema
  51. pharyngitis
  52. proteinuria
  53. pruritus
  54. rash
  55. respiratory arrest
  56. restlessness
  57. rhinitis
  58. rhinorrhea
  59. seizures
  60. serious hypersensitivity reactions or anaphylaxis
  61. sinus tachycardia
  62. skin necrosis
  63. skin ulcer
  64. supraventricular tachycardia (SVT)
  65. tachypnea
  66. tremor
  67. urticaria
  68. ventricular fibrillation
  69. ventricular tachycardia
  70. vomiting
  71. wheezing

Monitoring Parameters

  • LFTs

Contraindications

  • alglucosidase alfa hypersensitivity
  • breast-feeding
  • cardiac disease
  • cardiomyopathy
  • children
  • geriatric
  • heart failure
  • infants
  • infusion-related reactions
  • neonates
  • pregnancy
  • respiratory insufficiency
  • sepsis
  • serious hypersensitivity reactions or anaphylaxis
  • surgery

Interactions

There are no drug interactions associated with Alglucosidase alfa products.

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