Deflazacort

Medical uses

The manufacturer lists the following uses for deflazacort:

• Acute interstitial nephritis
• Anaphylaxis
• Asthma
• Autoimmune haemolytic anaemia
• Bullous pemphigoid
• Mixed connective tissue disease (other than systemic sclerosis)
• Crohn's disease
• Dermatomyositis
• Idiopathic thrombocytopenic purpura
• Juvenile chronic arthritis
• Severe hypersensitivity reactions
• Immunosuppression in transplantation
• Acute and lymphatic leukaemia
• Malignant lymphoma
• Multiple myeloma
• Muscular dystrophy
• Rheumatoid arthritis
• Polymyalgia rheumatica
• Nephrotic syndrome
• Pemphigus
• Polyarteritis nodosa
• Pyoderma gangrenosum
• Sarcoidosis
• Systemic lupus erythematosus
• Ulcerative colitis In the United States, deflazacort is approved for the treatment of duchenne muscular dystrophy in people over the age of two.

Adverse effects

Deflazacort carries the risks common to all corticosteroids, including immune suppression, decreased bone density, steroid induced muscle atrophy, myopathy and endocrine insufficiency. In clinical trials, the most common side effects (10% above placebo) were Cushing's-like appearance, weight gain, and increased appetite.

Pharmacology

Main article: Glucocorticoid#Pharmacology

Mechanism of action

Deflazacort is an inactive prodrug which is metabolized rapidly to the active drug 21-desacetyldeflazacort.

Relative potency

Deflazacort's potency is around 70–90% that of prednisone. A 2017 review found its activity of 7.5 mg of deflazacort is approximately equivalent to 25 mg cortisone, 20 mg hydrocortisone, 5 mg of prednisolone or prednisone, 4 mg of methylprednisolone or triamcinolone, or 0.75 mg of betamethasone or dexamethasone. The review noted that the drug has a high therapeutic index, being used at initial oral doses ranging from 6 to 90 mg, and probably requires a 50% higher dose to induce the same demineralizing effect as prednisolone. Thus it has "a smaller impact on calcium metabolism than any other synthetic corticosteroid, and therefore shows a lower risk of growth rate retardation in children and of osteoporosis" in the elderly, and comparatively small effects on carbohydrate metabolism, sodium retention, and hypokalemia.

History

Deflazacort was first introduced in 1969 to treat rheumatoid arthritis, nephritic syndrome, SLE, transplantation, polymyalgia rheumatica, sarcoidosis and juvenile chronic arthritis.

In January 2015, the US Food and Drug Administration (FDA) granted fast track status to Marathon Pharmaceuticals to pursue approval of deflazacort as a potential treatment for Duchenne muscular dystrophy, a rare, "progressive and fatal disease" that affects boys.

Society and culture

Legal status

The US Food and Drug Administration approved deflazacort to treat people aged five years of age and older with Duchenne muscular dystrophy, a rare genetic disorder that causes progressive muscle deterioration and weakness. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system. NDA 208684 was approved in February 2017, as a Type 1- new molecular entity with orphan status.

Economics

Deflazacort is available as an inexpensive generic. For example, in Canada, deflazacort can be purchased for around $1 per tablet.

Brand names

Deflazacort is sold in the United States under the brand name Emflaza after PTC Therapeutics, Inc. acquired all rights to Emflaza in March 2017. Deflazacort is sold in the United Kingdom under the trade name Calcort; in Brazil as Cortax, Decortil, Defcort and Deflanil; in India as Moaid, Zenflav, Defolet, DFZ, Decotaz, and DefZot; in Bangladesh as Xalcort; in Panama as Zamen; Spain as Zamene; and in Honduras as Flezacor.

References

References

1. (18 June 2024). "Emflaza- deflazacort tablet; Emflaza- deflazacort suspension".
2. (December 19, 2008). "Deflazacort versus other glucocorticoids: a comparison". Indian Journal of Dermatology.
3. (2006). "Analogue-based Drug Discovery". John Wiley & Sons.
4. (January 2018). "New Drug Therapy Approvals 2017".
5. "Refla: deflazacort".
6. "FDA approves drug to treat Duchenne muscular dystrophy".
7. (July 1995). "Pharmacokinetic/pharmacodynamic evaluation of deflazacort in comparison to methylprednisolone and prednisolone". Pharmaceutical Research.
8. (January 2017). "Deflazacort: therapeutic index, relative potency and equivalent doses versus other corticosteroids". BMC Pharmacology & Toxicology.
9. (2017-02-10). "Marathon Pharmaceuticals to Charge $89,000 for Muscular Dystrophy Drug".
10. (February 13, 2017). "Marathon Pharmaceuticals to Charge $89,000 for Muscular Dystrophy Drug After 70-Fold Increase".
11. "Drugs@FDA: FDA Approved Drug Products".
12. (February 10, 2017). "Brainstorm Health Daily".
13. "PTC Therapeutics Completes Acquisition of Emflaza for the Treatment of Duchenne Muscular Dystrophy in the U.S.".
14. (June 11, 2008). "Calcort". electronic Medicines Compendium.