Deflazacort

The manufacturer lists the following uses for deflazacort:{{cite web | url = http://julpharbd.com/products/pdf/1502065105738588954.pdf| title = Refla: deflazacort }}

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• 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

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In the United States, deflazacort is approved for the treatment of duchenne muscular dystrophy in people over the age of two.{{cite press release |title=FDA approves drug to treat Duchenne muscular dystrophy|url=https://www.fda.gov/news-events/press-announcements/fda-approves-drug-treat-duchenne-muscular-dystrophy |archive-url=https://web.archive.org/web/20190530140846/https://www.fda.gov/news-events/press-announcements/fda-approves-drug-treat-duchenne-muscular-dystrophy |url-status=dead |archive-date=30 May 2019 |website=U.S. Food and Drug Administration (FDA) |access-date=18 February 2017 }} {{PD-notice}}

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.

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Deflazacort is an inactive prodrug which is metabolized rapidly to the active drug 21-desacetyldeflazacort.{{cite journal | vauthors = Möllmann H, Hochhaus G, Rohatagi S, Barth J, Derendorf H | title = Pharmacokinetic/pharmacodynamic evaluation of deflazacort in comparison to methylprednisolone and prednisolone | journal = Pharmaceutical Research | volume = 12 | issue = 7 | pages = 1096–100 | date = July 1995 | pmid = 7494809 | doi = 10.1023/a:1016287104656 | s2cid = 9920545 }}

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.{{cite journal | vauthors = Parente L | title = Deflazacort: therapeutic index, relative potency and equivalent doses versus other corticosteroids | journal = BMC Pharmacology & Toxicology | volume = 18 | issue = 1 | pages = 1 | date = January 2017 | pmid = 28057083 | pmc = 5216559 | doi = 10.1186/s40360-016-0111-8 | doi-access = free }}

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.

{{citation|quote= has been shown to prolong lives ... a progressive and fatal disease that has no drug treatment available in the US|title=Duchenne muscular dystrophy drug could get OK for U.S. sales in 2016

|url=http://www.chicagotribune.com/business/ct-marathon-muscular-dystrophy-drug-0119-biz-20150119-story.html#page=1|publisher=The Chicago Tribune| vauthors = Hirst EJ |date= January 19, 2015|access-date=February 13, 2017 }} Although deflazacort was approved by the FDA for use in treatment of Duchenne muscular dystrophy on February 9, 2017,{{cite web|url=https://www.wsj.com/articles/marathon-pharmaceuticals-to-charge-89-000-for-muscular-dystrophy-drug-1486738267|title=Marathon Pharmaceuticals to Charge $89,000 for Muscular Dystrophy Drug|date=2017-02-10|website=The Wall Street Journal|archive-url=https://web.archive.org/web/20170210155321/https://www.wsj.com/articles/marathon-pharmaceuticals-to-charge-89-000-for-muscular-dystrophy-drug-1486738267|archive-date=2017-02-10|url-status=dead|access-date=2017-02-10}} Marathon CEO announced on February 13, 2017, that the launch of deflazacort (Emflaza) would be delayed amidst controversy over the steep price Marathon was asking for the drug in the United States - $89,000 per year, which is "roughly 70 times" more than it would cost overseas.{{citation |quote=FDA-approved deflazacort treats rare type of disease affecting boys |title=Marathon Pharmaceuticals to Charge $89,000 for Muscular Dystrophy Drug After 70-Fold Increase |url=https://www.wsj.com/articles/marathon-pharmaceuticals-to-charge-89-000-for-muscular-dystrophy-drug-1486738267|website=The Wall Street Journal | vauthors = Walker J, Pulliam S |date= February 13, 2017 |access-date=February 13, 2017 }}

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.{{cite web|url=https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&varApplNo=208684|title=Drugs@FDA: FDA Approved Drug Products|website=U.S. Food and Drug Administration (FDA)}}

Deflazacort is available as an inexpensive generic. For example, in Canada, deflazacort can be purchased for around $1 per tablet.{{cite web | url = http://fortune.com/2017/02/10/brainstorm-health-daily-02-10/ | title = Brainstorm Health Daily | date = February 10, 2017 |access-date= February 13, 2017 | vauthors = Mukherjee CS }}

Deflazacort is sold in the United States under the brand name Emflaza after PTC Therapeutics, Inc. acquired all rights to Emflaza in March 2017.{{cite web|url=http://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-completes-acquisition-emflazatm-treatment|title=PTC Therapeutics Completes Acquisition of Emflaza for the Treatment of Duchenne Muscular Dystrophy in the U.S.|website=PTC Therapeutics, Inc.}} Deflazacort is sold in the United Kingdom under the trade name Calcort;{{cite web | url = http://emc.medicines.org.uk/emc/assets/c/html/displaydoc.asp?documentid=20915 | archive-url = https://archive.today/20121224012214/http://emc.medicines.org.uk/emc/assets/c/html/displaydoc.asp?documentid=20915 | url-status = dead | archive-date = December 24, 2012 | title = Calcort | date = June 11, 2008 | publisher = electronic Medicines Compendium }} Retrieved on October 28, 2008. 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.

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{{Other drugs for disorders of the musculo-skeletal system}}

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Category:Acetate esters

Category:Corticosteroid cyclic ketals

Category:Corticosteroid esters

Category:Glucocorticoids

Category:Oxazolines

Category:Pregnanes

Category:Prodrugs

Category:Muscle protectors