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| Clinical data | |
|---|---|
| Trade names | Emflaza, Calcort, others |
| AHFS/Drugs.com | Monograph |
| Routes of administration | By mouth |
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| Legal status | |
| Pharmacokinetic data | |
| Protein binding | 40% |
| Metabolism | By plasmaesterases, to active metabolite |
| Eliminationhalf-life | 1.1–1.9 hours (metabolite) |
| Excretion | Kidney (70%) and fecal (30%) |
| Identifiers | |
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| PubChemCID | |
| DrugBank |
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| ChemSpider |
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| UNII | |
| KEGG |
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| ChEMBL | |
| CompTox Dashboard(EPA) | |
| ECHA InfoCard | 100.034.969 |
| Chemical and physical data | |
| Formula | C25H31NO6 |
| Molar mass | 441.524 g·mol−1 |
| 3D model (JSmol) | |
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Deflazacort, sold under the brand nameCalcort among others, is aglucocorticoid belonging to acetonides or O-isopropylidene derivative.[2] It is used as ananti-inflammatory and was patented in 1969[2] and approved for medical use in 1985.[3] The USFood and Drug Administration considers it to be afirst-in-class medication forDuchenne Muscular Dystrophy.[4]
The manufacturer lists the following uses for deflazacort:[5]
In the United States, deflazacort is approved for the treatment ofduchenne muscular dystrophy in people over the age of two.[6]
Deflazacort carries the risks common to allcorticosteroids, includingimmune suppression, decreasedbone density, steroid inducedmuscle atrophy,myopathy andendocrine insufficiency. In clinical trials, the most common side effects (>10% above placebo) wereCushing's-like appearance, weight gain, and increased appetite.[1]
Deflazacort is an inactiveprodrug which ismetabolized rapidly to the active drug 21-desacetyldeflazacort.[7]
Deflazacort's potency is around 70–90% that ofprednisone.[8] A 2017 review found its activity of 7.5 mg of deflazacort is approximately equivalent to 25 mgcortisone, 20 mghydrocortisone, 5 mg ofprednisolone orprednisone, 4 mg ofmethylprednisolone ortriamcinolone, or 0.75 mg ofbetamethasone ordexamethasone. The review noted that the drug has a hightherapeutic 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.[9]
Deflazacort was first introduced in 1969 to treat rheumatoid arthritis, nephritic syndrome, SLE, transplantation, polymyalgia rheumatica, sarcoidosis and juvenile chronic arthritis.[2]
In January 2015, the USFood and Drug Administration (FDA) grantedfast track status toMarathon Pharmaceuticals to pursue approval of deflazacort as a potential treatment forDuchenne muscular dystrophy, a rare, "progressive and fatal disease" that affects boys.[10] Although deflazacort was approved by the FDA for use in treatment of Duchenne muscular dystrophy on February 9, 2017,[6][11] 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.[12]
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.[6] NDA 208684 was approved in February 2017, as a Type 1- new molecular entity with orphan status.[13]
Deflazacort is available as an inexpensive generic. For example, in Canada, deflazacort can be purchased for around $1 per tablet.[14]
Deflazacort is sold in the United States under the brand name Emflaza after PTC Therapeutics, Inc. acquired all rights to Emflaza in March 2017.[15] Deflazacort is sold in the United Kingdom under the trade name Calcort;[8] 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.
This article incorporates text from this source, which is in thepublic domain.has been shown to prolong lives ... a progressive and fatal disease that has no drug treatment available in the US
FDA-approved deflazacort treats rare type of disease affecting boys
