Deflazacort

Deflazacort Brand Name– EMFLAZA

What is Deflazacort

Deflazacort is an oral corticosteroid indicated for the treatment of patients 2 years of age and older with Duchenne muscular dystrophy (DMD).

DMD is the most common type of muscular dystrophy and is caused by an absence of dystrophin, a protein that helps keep muscle cells intact.

Affected patients typically develop symptoms in early childhood (usually between 3 and 5 years of age) that quickly progress to a loss of ambulation in adolescence and ultimately, life-threatening heart and respiratory conditions that limit life expectancy to early adulthood.

The disease primarily affects boys, but in rare cases it can affect girls. DMD affects approximately 1 in 3,600 male births worldwide.

Corticosteroids are commonly used to treat DMD worldwide, but deflazacort is the first corticosteroid approved for DMD treatment in the United States.

This approval was based on the effectiveness of deflazacort in a clinical trial involving 196 male patients 5 to 15 years of age at the beginning of the trial with documented mutation of the dystrophin gene and onset of weakness before age 5.

At 12 weeks, patients receiving deflazacort had improvements in average strength of 18 muscle groups.

The persistence of the treatment effect was also documented in the deflazacort group at week 52. In another trial lasting 104 weeks and involving 29 male patients, deflazacort demonstrated a numerical advantage over placebo on an assessment of average muscle strength.

The most common adverse reactions reported during clinical trials were similar to those experienced with other corticosteroids and included Cushingoid appearance, weight again, increased appetite, upper respiratory tract infections, cough, pollakiuria, hirsutism, and central obesity.

Indications

• Duchenne muscular dystrophy

For the treatment of Duchenne muscular dystrophy (DMD)

NOTE: The FDA has designated deflazacort as an orphan drug for this indication.

Side Effects

1. abdominal pain
2. adrenocortical insufficiency
3. alopecia
4. amnesia
5. anaphylactoid reactions
6. anxiety
7. appetite stimulation
8. avascular necrosis
9. back pain
10. bone fractures
11. candidiasis
12. cataracts
13. confusion
14. constipation
15. cough
16. Cushing’s syndrome
17. depression
18. diabetes mellitus
19. dizziness
20. dyspepsia
21. dysuria
22. edema
23. emotional lability
24. epistaxis
25. erythema
26. fever
27. GI perforation
28. glycosuria
29. growth inhibition
30. hallucinations
31. heart failure
32. hirsutism
33. hyperactivity
34. hyperglycemia
35. hypertension
36. hypoventilation
37. impaired wound healing
38. increased intracranial pressure
39. infection
40. influenza
41. insomnia
42. irritability
43. lacrimation
44. leukocytosis
45. mania
46. myalgia
47. myopathy
48. nausea
49. pancreatitis
50. peptic ulcer
51. pharyngitis
52. pseudotumor cerebri
53. psychosis
54. rash
55. rhinorrhea
56. seizures
57. striae
58. suicidal ideation
59. tendon rupture
60. testicular pain
61. thromboembolism
62. toxic epidermal necrolysis
63. vertigo
64. weakness
65. weight gain
66. withdrawal

Monitoring Parameters

• blood glucose
• blood pressure
• growth rate
• ophthalmologic exam
• serum potassium
• weight

Contraindications

• abrupt discontinuation
• breast-feeding
• cataracts
• corticosteroid hypersensitivity
• Cushing’s syndrome
• diabetes mellitus
• diverticulitis
• fungal infection
• GI disease
• GI perforation
• glaucoma
• growth inhibition
• heart failure
• hepatitis
• hypertension
• hyperthyroidism
• hypothalamic-pituitary-adrenal (HPA) suppression
• hypothyroidism
• infants
• infection
• measles
• myasthenia gravis
• myocardial infarction
• neonates
• osteoporosis
• peptic ulcer disease
• pheochromocytoma
• pregnancy
• psychosis
• renal disease
• sepsis
• thromboembolic disease
• ulcerative colitis
• vaccination
• varicella

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