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.