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EMFLAZA deflazacort 36 mg Tablet, 30-count — NDC 52856-0504-03 package photo

EMFLAZA deflazacort 36 mg Tablet, 30-count

by PTC Therapeutics, Inc. · 30 TABLET in 1 BOTTLE (52856-504-03)
NDC 52856-0504-03
🏷️ FDA NDC (as labeled) 52856-504-03 billing pads the product segment with a zero
Rx only Brand On market Non-controlled
🗂️ Data synced Jul 24, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

This page is curated, coded, and maintained by

Compiled from public FDA, CMS, DailyMed, NADAC, and related drug data sources. Editorial policy

🆔 Identity & classification

FDA NDC (as labeled) 52856-504-03
Product NDC 52856-504
11-digit billing NDC 52856050403
NCPDP billing unit EA — each (per item)
RxCUI 153098, 153880, 1870950, 1870952, 1870954, 1870956, 1870958, 1870960,
UNII KR5YZ6AE4B
Application # NDA208684
SPL Set ID 31b347d2-f156-4055-9d8f-7cf0df420296
Established class (EPC) Corticosteroid
Mechanism of action Corticosteroid Hormone Receptor Agonists
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2018-09-10
Route ORAL
Dosage form TABLET
Substance DEFLAZACORT
GPI-14 22100017000365
GPI class Emflaza
GCN Seq No 077116
GCN 43015
HICL code 011668
Ingredient (HICL) Deflazacort
HIC1 code P
Therapeutic class — broad (HIC1) Endocrine System
HIC2 code P5
Therapeutic class — intermediate (HIC2) Adrenocortical Hormones
HIC3 code P5A
Therapeutic class — specific (HIC3) Glucocorticoids
AHFS code 68:04.00.00
AHFS class Adrenals
FDB label name EMFLAZA 36 MG TABLET
FDB brand name Emflaza
Legend status F — Federal legend — prescription drug or device
TE code (Orange Book) AB · RLD · RS
Why two NDCs? The FDA registers this code as 52856-504-03 — a 5-3-2 layout, and that's what's printed on the package and shown on DailyMed. For insurance claims, every NDC is standardized to a uniform 11-digit 5-4-2 format by adding a zero to the product segment → 52856-0504-03. Same drug, same package — only the format differs.
Where does this data come from?
Identifiers from the FDA openFDA NDC Directory and Structured Product Labeling; RxCUI from RxNorm (NLM); GPI from Medi-Span; GCN / HIC / AHFS / legend from First Databank.

🏷️ RxNorm drug class

This medicine belongs to the Corticosteroid class.

Pharmacologic class Corticosteroid
Drug family (ATC) Glucocorticoids
How it works Corticosteroid Hormone Receptor Agonists
Where does this data come from?
Therapeutic classes from RxNorm RxClass (U.S. National Library of Medicine) — Established Pharmacologic Class (FDA), ATC drug family (WHO) and mechanism of action, matched by this product’s RxCUI.

🏭 Manufacturer & labeler

LabelerPTC Therapeutics, Inc.
Application holderPTC THERAPEUTICS INC
FDA applicationNDA208684 (NDA)
Labeler code52856
First marketedSep 2018
Product typeHuman Prescription Drug
Portfolio6 products on file
The labeler markets the product; the application holder owns the FDA approval. They’re often the same company but can differ (e.g. a repackager or an authorized generic). A mailing address / phone appears here when the manufacturer includes it in the product’s FDA label (not all do).
Where does this data come from?
Labeler, application holder and registered establishment from the FDA openFDA NDC Directory and Drugs@FDA; address/contact from the product’s FDA label.

🩺 Clinical

Label name EMFLAZA 36 MG TABLET Ingredient Deflazacort
📖 What it is MedlinePlus · NLM

Deflazacort is used to treat Duchenne muscular dystrophy (DMD; a progressive disease in which the muscles do not function properly). Deflazacort is in a class of medications called corticosteroids. It works by reducing inflammation (swelling) and by changing the way the immune system works.

Read the full MedlinePlus article ↗
📗 Our plain-language guide HelloPharmacist
  • Deflazacort is used to treat Duchenne muscular dystrophy (DMD), a genetic disease that causes muscles to weaken and break down over time. It's a corticosteroid — a type of anti-inf...
  • Yes — several brands, including Emflaza, Jaythari, and Kymbee tablets, can be crushed and mixed immediately with applesauce if swallowing a whole tablet is difficult. The oral susp...
  • Can my child take the tablet if they have trouble swallowing pills?
  • Many children on deflazacort develop a rounder, puffier face (sometimes called a 'moon face'), gain weight, grow more body hair, and get more frequent infections — these are common...
📖 Read our full Deflazacort guide →
Where does this data come from?
Plain-language summary from MedlinePlus (U.S. National Library of Medicine); supplement & herbal interactions and nutrient depletion data from the Natural Medicines database; our full guide is HelloPharmacist editorial content.

💊 What it looks like

Color White
ShapeOval
Imprint36
Size17 mm
ScoringNot scored
One label can cover several strengths, so colors may be combined — always confirm a loose pill against the dispensed prescription label or a pharmacist.
Where does this data come from?
Physical description (imprint, shape, color, scoring, coating) from this product’s FDA Structured Product Labeling (SPL), mirrored from DailyMed / openFDA.

🧪 Inactive Ingredients / Excipients

Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.

💡 Tap an ingredient (hover on desktop) to see what it is and why it’s used.

  • UNII EWQ57Q8I5X
    Lactose monohydrate is a natural sugar derived from milk. It serves as a filler and binder in tablets and capsules, helping create the proper size, texture, and consistency of the medicine.
  • UNII 70097M6I30
    Magnesium stearate is a salt made from magnesium and stearic acid, a fatty substance. It's used in tablets and capsules as a lubricant and glidant to help ingredients flow smoothly during manufacturing and prevent sticking.
  • UNII ETJ7Z6XBU4
    Silicon dioxide is a naturally occurring mineral used as a glidant and anti-caking agent. It helps powder ingredients flow smoothly and prevents clumping during manufacturing and storage.
  • UNII O8232NY3SJ
    A plant-based carbohydrate derived from corn kernels. It acts as a filler to add bulk, a binder to hold ingredients together, and a disintegrant to help the tablet break apart in your stomach for absorption.

4 inactive ingredients listed in the exact product block matched to this NDC.

Where does this data come from?
Data sourced from official FDA Structured Product Labeling (SPL) via DailyMed — ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.

Inactive ingredient FAQ

Are inactive ingredients the same for every manufacturer?
No. Inactive ingredients can differ by manufacturer, dosage form, strength, and package / product version.
Why might an inactive ingredient be missing?
Some SPLs do not provide a complete structured inactive-ingredient list, and older or unusual labels may only include the information in narrative text.
Can inactive ingredients matter?
Yes. They can matter for allergies, intolerances, dyes, gluten / lactose concerns, preservatives, and formulation differences — but confirm with a pharmacist or the manufacturer when it’s clinically important.

💲 Pricing

A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.

Price systemPer eachPer package
Retail pharmacies payNADAC · weekly Not in the retail survey — common for institutional, discontinued, or low-volume packs.
Medicaid paysCMS SDUD · 12 mo $590.01 $17,700.32 / 30 tablets
Medicare drug plans payPart D · quarterly No Part D plan price is available for this NDC in our data.
Where does this data come from?
NADAC (National Average Drug Acquisition Cost) is the CMS weekly pharmacy-acquisition-cost survey — what pharmacies pay. ASP (Average Sales Price) is the CMS Medicare Part B drug-payment file, published quarterly. Medicaid pays is computed by us from CMS State Drug Utilization Data (total reimbursed ÷ units, trailing 12 months) — gross of rebates and inclusive of dispensing fees, so it reflects what Medicaid paid, not an acquisition cost. Medicare drug plans pay is the median negotiated point-of-sale unit cost across plans listing this NDC in the CMS quarterly Prescription Drug Plan pricing files, before rebates. The VA pays is the federal contract price (FSS, and the statutory Big 4 ceiling where listed) from the VA National Acquisition Center pharmaceutical price file. All are free public government data; each measures a different payer, so the figures are not directly comparable.

🔁 Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Kymbee 36 mg 00245-0817-30 Upsher-Smith 30 tablets — AB FDA listed —
Deflazacort 36 mg 00832-0817-30 Upsher-Smith 30 tablets — AB FDA listed —
Emflaza 36 mgthis 52856-0504-03 PTC 30 tablets — AB FDA listed —
Deflazacort 36 mg 59651-0602-30 Aurobindo 30 tablets — AB FDA listed —
Deflazacort 36 mg 60219-2285-01 Amneal 30 tablets — AB FDA listed —
Deflazacort 36 mg 70095-0043-30 Sun 30 tablets — AB FDA listed —
Jaythari 36 mg 70710-1752-03 Zydus 30 tablets — AB FDA listed —
Deflazacort 36 mg 70710-2054-03 Zydus 30 tablets — AB FDA listed —
Deflazacort 36 mg 73190-0071-30 AvKARE 30 tablets — AB FDA listed —
Jaythari 36 mg 81806-0177-30 Doppel 30 tablets — AB FDA listed —
Deflazacort 36 mg 81806-0208-30 Doppel 30 tablets — AB FDA listed —
About this product: this is the brand-name version. Some generic versions are approved by the FDA, but we could not confirm current pharmacy availability from our pricing/market data.
Where does this data come from?
Equivalents are other NDCs of the same ingredient, form and route from the openFDA NDC Directory, ranked least-expensive-first by NADAC. Therapeutic-equivalence (AB) ratings come from the FDA Orange Book; biologics use the FDA Purple Book for biosimilar & interchangeable status.

⏳ Availability & generic status

🏛️
2017
First FDA approval
Feb 2017
📍
2026
Currently FDA-listed
9 years listed
🔓
·
Listed protection lapsed
listed protections lapsed 2026
🔒Generic approved by FDA, but pharmacy availability is not confirmed

The FDA lists approved generic versions of this medicine, but that does not always mean a pharmacy can get one today. Patent rules, launch agreements, supply and pricing can affect when generics actually arrive.

🛡️ Latest patent/protection date listed: The latest listed FDA patent/protection lapsed Jun 2026 — those protections no longer apply, though a generic still needs FDA approval and a manufacturer to market it.
📅 FDA approved Feb 9, 2017 AB TE-rated RLD RS

Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.

Patents & exclusivity — FDA Orange Book
Exclusivity ODE-252
Exclusivity ODE-252
Exclusivity ODE-252
Exclusivity ODE-252
2017 2019 2021 2023 2025
Today
LOE
Substance patent Formulation patent Method-of-use patent Exclusivity Pediatric +6mo
🏛️FDA exclusivity
FDA-granted marketing protection. It’s separate from patents and may be shorter than patent protection.
🧪Product / substance patents
Patents covering the active ingredient, product, formulation, or related drug features.
🎯Method-of-use patents
Patents covering specific approved uses. These can sometimes be carved out with a “skinny label,” but not always.
🛈 What do these terms mean?
Patent
Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
Substance patent
Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
Formulation (product) patent
Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
Method-of-use patent
A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
Skinny label
A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
Exclusivity
FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
Paragraph IV
A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
RLD / RS
Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
TE / AB rating
FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
LOE (loss of exclusivity)
The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.

Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.

FDA exclusivity
CodeWhat it grantsExpires
ODE-252Orphan Drug Exclusivity (7-year)Jun 7, 2026
ODE-252Orphan Drug Exclusivity (7-year)Jun 7, 2026
ODE-252Orphan Drug Exclusivity (7-year)Jun 7, 2026
ODE-252Orphan Drug Exclusivity (7-year)Jun 7, 2026
Common questions
Is there a generic version of EMFLAZA 36 MG TABLET?
Yes — an FDA-approved generic equivalent is listed in the FDA Orange Book for EMFLAZA 36 MG TABLET. See the alternatives section for substitutable, lower-cost products.
The FDA approved a generic — why can’t I get it at my pharmacy yet?
FDA approval and pharmacy availability are two different things. The FDA can approve a generic years before it actually reaches pharmacies, because the brand company may still hold patents or have a settlement that delays the launch. A manufacturer also has to choose to make and sell it, and have supply ready. So a drug can be “FDA-approved generic exists” and still be brand-only at the counter today.
Why do different websites show different generic release dates?
Generic availability is not based on one single date. Some sources use the first exclusivity expiration, some use the last product patent, and others use the latest method-of-use patent. Patent challenges, settlements, licenses, and label carve-outs can also change the real-world launch date. This page shows the underlying Orange Book dates so you can see why estimates may differ.
What does “FDA listed” mean?
It means the product appears in the FDA’s official NDC directory. That’s a good sign a product exists and is intended for the U.S. market, but on its own it does not confirm a pharmacy can fill it today. Where we have recent retail pricing data (NADAC) for a product, we label it “Availability likely” instead.
What does a patent or protection date mean here?
It’s the latest date currently listed in the FDA Orange Book for a patent or exclusivity on the brand product. It can affect when a full generic version becomes widely available — but it is not a guaranteed generic launch date. Generics sometimes arrive earlier (through a settlement or patent challenge) or later (a manufacturer still has to make and sell one).
What does “current Orange Book estimate” mean?
It means we are using the latest patent and exclusivity dates currently listed in the FDA Orange Book. It is not a guaranteed launch date.
Can a generic come out before the last patent expires?
Sometimes. A generic company may challenge a patent, settle with the brand manufacturer, receive a license, or obtain approval with a narrower label that avoids a patented use. In other cases, the last listed protection may delay full-label generic competition.
Can a generic come out after the listed dates?
Yes. Even after patents or exclusivity expire, a generic still needs FDA approval and a manufacturer must choose to market it. Supply, litigation, business decisions, or regulatory issues can delay actual availability.
What is the difference between patents and exclusivity?
Patents are legal protections usually issued by the U.S. Patent and Trademark Office. FDA exclusivity is marketing protection granted by the FDA. They are separate, and either one can affect generic timing.
Why are there multiple patent dates?
One drug can have several patents covering different things: the active ingredient, a formulation, a manufacturing process, or a specific approved use. That is why a page may show several expiration dates instead of one simple generic date.
Built from FDA Orange Book patent and exclusivity data. Dates are refreshed from public FDA data when available; the marker is max(latest patent expiry, latest exclusivity expiry). Paragraph-IV settlements and first-filer 180-day exclusivity can shift the real date; a method-of-use patent may allow an earlier skinny-label generic for non-protected indications. Generic launch timing is an estimate, not a guarantee.
Where does this data come from?
Patents and exclusivity from the FDA Orange Book (small-molecule drugs), refreshed from public FDA data. Generic launch timing is an estimate, not a guarantee.

🗺️ Medicaid utilization & spend

📍 This exact package only: Medicaid data is reported per full 11-digit NDC — labeler, product and pack size — so every number here is for 52856-0504-03, not the drug overall. Other pack sizes report separately.
💊 Pharmacy benefit only: These are Medicaid outpatient pharmacy claims, billed by NDC. They exclude the medical benefit — clinic- or hospital-administered drugs billed under HCPCS J-codes — so drugs used mostly that way (e.g. Avastin, Lucentis, Keytruda) can look low or missing here. That’s expected, not an error.
📅 Q1 2025 – Q1 2026 · 5 quarters of data
ⓘ The newest quarter is usually incomplete when first published; states restate recent quarters in later CMS releases, so the latest figures typically revise upward. State coverage-policy changes can also shift quarter-to-quarter totals.
Prescriptions last 4 qtrs
1.6K
Units reimbursed last 4 qtrs
45.2K
Gross reimbursed last 4 qtrs
$26.69M
Avg / prescription
$16,584.84
Avg / unit
$590.01
Latest quarter Q1 2026
376Rx
Fee-for-service vs managed care ⓘ
50% FFS 50% MCO
Fee-for-service · 799 Rx Managed care · 810 Rx
State Medicaid map
Alaska: no data reported AK Maine: no data reported ME Washington: no data reported WA Idaho: no data reported ID Montana: no data reported MT North Dakota: no data reported ND Minnesota: no data reported MN Wisconsin: 2,286 units · 38.7 per 100k residents WI Michigan: 2,515 units · 25.1 per 100k residents MI New York: 2,550 units · 13.0 per 100k residents NY Vermont: no data reported VT New Hampshire: no data reported NH Oregon: no data reported OR Nevada: no data reported NV Wyoming: no data reported WY South Dakota: no data reported SD Iowa: no data reported IA Illinois: 1,138 units · 9.1 per 100k residents IL Indiana: 2,925 units · 42.6 per 100k residents IN Ohio: 2,062 units · 17.5 per 100k residents OH Pennsylvania: no data reported PA New Jersey: no data reported NJ Massachusetts: 1,710 units · 24.4 per 100k residents MA California: 7,354 units · 18.9 per 100k residents CA Utah: no data reported UT Colorado: 1,280 units · 21.8 per 100k residents CO Nebraska: no data reported NE Missouri: no data reported MO Kentucky: no data reported KY West Virginia: 1,305 units · 73.7 per 100k residents WV Virginia: no data reported VA Maryland: no data reported MD Connecticut: no data reported CT Rhode Island: no data reported RI Arizona: 1,899 units · 25.6 per 100k residents AZ New Mexico: no data reported NM Kansas: no data reported KS Arkansas: 3,014 units · 98.3 per 100k residents AR Tennessee: no data reported TN North Carolina: 3,172 units · 29.3 per 100k residents NC South Carolina: no data reported SC Delaware: no data reported DE Oklahoma: no data reported OK Louisiana: no data reported LA Mississippi: 450 units · 15.3 per 100k residents MS Alabama: no data reported AL Georgia: 1,950 units · 17.7 per 100k residents GA D.C.: no data reported DC Hawaii: no data reported HI Texas: 6,842 units · 22.4 per 100k residents TX Florida: 2,776 units · 12.3 per 100k residents FL
Units reimbursed · per 100k residents
9.198.3
gray = no data reported ⓘ
Colors are per 100,000 residents, so big states don’t automatically dominate. Tap or hover a state for its actual totals.
Tap or hover a state
…for its Medicaid breakdown
🏆 Top states by units · per 100k residents
1 Arkansas 98.3 /100k
2 West Virginia 73.7 /100k
3 Indiana 42.6 /100k
4 Wisconsin 38.7 /100k
5 North Carolina 29.3 /100k
6 Arizona 25.6 /100k
7 Michigan 25.1 /100k
8 Massachusetts 24.4 /100k
National units — by quarter
💵 About the dollar figures: “reimbursed” is what Medicaid paid pharmacies before confidential manufacturer rebates, so the program’s real net cost is lower than these numbers. Fee-for-service and managed-care claims are combined unless split above. Source: CMS State Drug Utilization Data; per-100k rates use 2023 Census population estimates.

📊 Medicare Part D spend CMS · PART D · 2026 (Q1)

Medicare Part D (outpatient prescription) spending for Emflaza — the program that covers self-administered drugs. 1 manufacturer.
⚠️ Drug-level data: CMS publishes Part D spending by drug, not by NDC — these figures combine every manufacturer, strength and package size sold under the name Emflaza. That’s a different level of aggregation than the Medicaid card above, which is specific to this exact 11-digit NDC (pack size included), so the two aren’t directly comparable.
Period
Total Part D spend
$3.34M
Claims incl. refills
243
Beneficiaries
75
Spend / beneficiary
$44,482.27
Spend / claim
$13,729.10
Trend by period
💵 About the dollar figures: spending is what Part D plans paid before confidential manufacturer rebates, so the program’s real net cost is lower. A blank patient count means fewer than 11 people — CMS hides counts that small to protect privacy. Source: CMS Medicare Quarterly Part D Spending by Drug (data.cms.gov), updated quarterly.

🔬 Reported adverse events (FAERS)

Read carefully: FAERS reports are voluntary and unverified. Counts are not incidence, do not establish causation, are subject to reporting bias, and cannot be used to compare one drug to another. Shown for signal context only. Reports for EMFLAZA (this brand).

Top reported reactions

Weight Increased531
Fall300
Vomiting256
Pain228
Pyrexia227
Headache219
Malaise216

Age at onset

Neonate2
Infant4
Child29
Adolescent3
Adult209
Elderly112

Reporter sex

6,355 reports
Male · 66%
Female · 34%
Unknown · 0%

Serious outcomes

Hospitalization1,576
Death306
Life-threatening183
Disabling120
Reports over time (by year) — tap or hover for the count & year
2019 2021 2023 2026 1,635 0
Most recent year is provisional (FAERS lags ~3 months).
Where does this data come from?
Adverse-event reports from the FDA Adverse Event Reporting System (FAERS) via openFDA. FAERS reports are voluntary and unverified — counts are not incidence and don’t establish causation.

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
52856-0504-03 You're viewing this 30 TABLET in 1 BOTTLE (52856-504-03) 2018-09-10 Active

🧭 About this NDC listing & data coverage

Finished prescription product
What data is (and isn’t) available for this NDC — tap to expand
NDC identity (package / product / labeler codes) ✓ Available
Labeler ✓ Available
Product & package description ✓ Available
Marketing category & status ✓ Available
Active ingredient / dosage form / route ✓ Available
FDA label (SPL via DailyMed) ✓ Available
Package photos ✓ Available
Inactive ingredients (structured) ✓ Available
NADAC pharmacy acquisition price (CMS) — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey.
Orange Book / therapeutic-equivalence data ✓ Available
HCPCS J-code billing crosswalk — Not published for this NDC Most self-administered / retail products have no J-code — that is normal.
Medicaid utilization (CMS SDUD) ✓ Available
“Not published” reflects what the public FDA / CMS / NLM sources provide for this exact package code — it is a property of the data feeds, not a judgment about the product.

Questions about this listing

Why is there no price listed?
The pricing shown on our NDC pages comes from CMS NADAC, a voluntary survey of retail community pharmacy invoices. CMS does not publish a NADAC for every NDC — packages outside the retail survey (institutional and hospital products, bulk packages, discontinued items, and many OTC items) may never receive one. A missing price reflects the survey's scope, not this product's actual cost, and does not mean the product is free or unavailable.
Is the NDC printed on the package the same as the 11-digit billing NDC?
Yes, they identify this exact package in different formats. The FDA registers it as 52856-504-03, which is what is printed on the packaging and shown on DailyMed. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero: 52856-0504-03, written without dashes as 52856050403. The Identity section at the top of this page lists every form of this code.
What do the three segments of this NDC mean?
In 52856-0504-03, the first segment (52856) is the labeler code FDA assigned to PTC Therapeutics, Inc.; the middle segment (0504) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (03) identifies this exact package size and type. Together they name one specific package of one specific product.
Is this package still being marketed?
Yes, per the latest FDA NDC Directory data on this page: this package is listed as actively marketed, with no marketing end date reported by PTC Therapeutics, Inc.. Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
PTC Therapeutics, Inc. is the labeler of record for this NDC — the company under whose FDA-assigned code the package is listed. The labeler may be the manufacturer itself or a distributor marketing the product under its own code.
Do I need a prescription for this product?
This NDC is listed with FDA as a prescription product, so it is dispensed under a prescriber's order. Your pharmacist can tell you whether any over-the-counter forms of the same medication exist.
This page identifies an FDA-listed package (the NDC) and reports public regulatory and pricing data about the listing. It is reference information, not a medical recommendation — talk to your pharmacist or prescriber about your own medication.
Where does this data come from?
Listing facts (marketing category, packager status, marketing dates) from the FDA openFDA NDC Directory; label availability from DailyMed; pricing coverage from CMS NADAC; equivalence scope from the FDA Orange Book.

📄 Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Jump with a chip, search within the label, or expand everything.
🎯 Indications and Usage ⏱️ Dosage and Administration 💊 Dosage Forms and Strengths ⛔ Contraindications ⚠️ Warnings and Cautions 🤒 Adverse Reactions 🔄 Drug Interactions 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🆘 Overdosage 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📦 Storage and Handling 📋 Description 💬 Information for Patients
🎯 Indications and Usage 47 words ▾

1 INDICATIONS AND USAGE EMFLAZA is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. EMFLAZA is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older ( 1 )

⏱️ Dosage and Administration ~2 min read ▾

2 DOSAGE AND ADMINISTRATION The recommended once-daily dosage is approximately 0.9 mg/kg/day administered orally ( 2.2 ) Discontinue gradually when administered for more than a few days ( 2.3 )

2.1Assessments Prior to First Dose of EMFLAZA Administer all immunizations according to immunization guidelines prior to starting EMFLAZA. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting EMFLAZA [see Warnings and Precautions ( 5.8 )] .

2.2Dosing Information The recommended oral dosage of EMFLAZA is approximately 0.9 mg/kg/day once daily. If tablets are used, round up to the nearest possible dose. Any combination of the four EMFLAZA tablet strengths can be used to achieve this dose. If the oral suspension is used, round up to the nearest tenth of a milliliter (mL).

2.3Discontinuation Dosage of EMFLAZA must be decreased gradually if the drug has been administered for more than a few days [see Warnings and Precautions ( 5.1 )] .

2.4Important Preparation and Administration Instructions EMFLAZA Tablets and Oral Suspension can be taken with or without food. Do not administer EMFLAZA with grapefruit juice [see Drug Interactions ( 7.1 )] EMFLAZA Tablets EMFLAZA Tablets can be administered whole or crushed and taken immediately after mixing with applesauce. EMFLAZA Oral Suspension Shake EMFLAZA Oral Suspension well before administration.

Use only the oral dispenser provided with the product. After withdrawing the appropriate dose into the oral dispenser, slowly add the EMFLAZA Oral Suspension into 3 to 4 ounces of juice (except grapefruit juice) or milk and mix well. The dose should then be administered immediately.

Discard any unused EMFLAZA Oral Suspension remaining after 1 month of first opening the bottle.

2.5Dosage Modification for Use with CYP3A4 Inhibitors and Inducers CYP3A4 Inhibitors Give one third of the recommended dosage when EMFLAZA is administered with moderate or strong CYP3A4 inhibitors. For example, a 36 mg per day dose would be reduced to a 12 mg per day dose when used with moderate or strong CYP3A4 inhibitors [see Drug Interactions ( 7.1 ) and Clinical Pharmacology ( 12.3 )] . CYP3A4 Inducers Avoid use with moderate or strong CYP3A4 inducers with EMFLAZA [see Drug Interactions ( 7.1 ) and Clinical Pharmacology ( 12.3 )] .

💊 Dosage Forms and Strengths 76 words ▾

3 DOSAGE FORMS AND STRENGTHS Tablets 6 mg: White and round with “6” debossed on one side 18 mg: White and round with “18” debossed on one side 30 mg: White and oval with “30” debossed on one side 36 mg: White and oval with “36” debossed on one side Oral Suspension 22.75 mg/mL: Whitish suspension Tablets: 6 mg, 18 mg, 30 mg, and 36 mg ( 3 ) Oral Suspension: 22.75 mg/mL ( 3 )

⛔ Contraindications 59 words ▾

4 CONTRAINDICATIONS EMFLAZA is contraindicated in patients with known hypersensitivity to deflazacort or to any of the inactive ingredients. Instances of hypersensitivity, including anaphylaxis, have occurred in patients receiving corticosteroid therapy [see Warnings and Precautions ( 5.15 ) and Adverse Reactions ( 6.2 )] . Hypersensitivity to deflazacort or any of the inactive ingredients in EMFLAZA ( 4 )

⚠️ Warnings and Cautions ~2 min read ▾

5 WARNINGS AND PRECAUTIONS Alterations in Endocrine Function: Hypothalamic-pituitary-adrenal axis suppression, Cushing’s syndrome, and hyperglycemia can occur; Monitor patients for these conditions with chronic use of EMFLAZA ( 2.3 , 5.1 ) Immunosuppression and Increased Risk of Infection: Increased risk of new, exacerbation, dissemination, or reactivation of latent infections, which can be severe and at times fatal; Signs and symptoms of infection may be masked ( 5.2 ) Alterations in Cardiovascular/Renal Function: Monitor for elevated blood pressure and sodium, and for decreased potassium levels ( 5.3 ) Gastrointestinal Perforation: Increased risk in patients with certain GI disorders; Signs and symptoms may be masked ( 5.4 ) Behavioral and Mood Disturbances: May include euphoria, insomnia, mood swings, personality changes, severe depression, and psychosis ( 5.5 ) Effects on Bones: Monitor for decreases in bone mineral density with chronic use of EMFLAZA ( 5.6 ) Ophthalmic Effects: May include cataracts, infections, and glaucoma; Monitor intraocular pressure if EMFLAZA is continued for more than 6 weeks ( 5.7 ) Vaccination: Do not administer live or live attenuated vaccines to patients receiving immunosuppressive doses of corticosteroids.

Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting EMFLAZA ( 5.8 ) Serious Skin Rashes: Discontinue at the first sign of rash, unless the rash is clearly not drug related ( 5.9 )

5.1Alterations in Endocrine Function Corticosteroids, such as EMFLAZA, can cause serious and life-threatening alterations in endocrine function, especially with chronic use. Monitor patients receiving EMFLAZA for Cushing’s syndrome, hyperglycemia, and adrenal insufficiency after EMFLAZA withdrawal. In addition, patients with hypopituitarism, primary adrenal insufficiency or congenital adrenal hyperplasia, altered thyroid function, or pheochromocytoma may be at increased risk for adverse endocrine events.

Risk of Adrenal Insufficiency Following Corticosteroid Withdrawal Corticosteroids produce reversible hypothalamic-pituitary-adrenal (HPA) axis suppression, with the potential for the development of secondary adrenal insufficiency after withdrawal of corticosteroid treatment. Acute adrenal insufficiency can occur if corticosteroids are withdrawn abruptly, and can be fatal. The degree and duration of adrenocortical insufficiency produced is variable among patients and depends on the dose, frequency, and duration of corticosteroid therapy.

The risk is reduced by gradually tapering the corticosteroid dose when withdrawing treatment. This insufficiency may persist, however, for months after discontinuation of prolonged therapy; therefore, in any situation of stress occurring during that period of discontinuation, corticosteroid therapy should be reinstituted. For patients already taking corticosteroids during times of stress, the dosage may need to be increased.

A steroid “withdrawal syndrome”, seemingly unrelated to adrenocortical insufficiency, may also occur following abrupt discontinuance of corticosteroids. This syndrome includes symptoms such as anorexia, nausea, vomiting, lethargy, headache, fever, joint pain, desquamation, myalgia, and/or weight loss. These effects are thought to be due to the sudden change in corticosteroid concentration rather than to low corticosteroid levels.

Cushing’s Syndrome Cushing’s syndrome (hypercortisolism) occurs with prolonged exposure to exogenous corticosteroids, including EMFLAZA. Symptoms include hypertension, truncal obesity and thinning of the limbs, purple striae, facial rounding, facial plethora, muscle weakness, easy and frequent bruising with thin fragile skin, posterior neck fat deposition, osteopenia, acne, amenorrhea, hirsutism and psychiatric abnormalities. Hyperglycemia Corticosteroids can increase blood glucose, worsen pre-existing diabetes, predispose those on long-term therapy to diabetes mellitus, and may reduce the effect of anti…

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The following serious adverse reactions are discussed in more detail in other sections: Alterations in Endocrine Function [see Warnings and Precautions ( 5.1 )] Immunosuppression and Increased Risk of Infection [see Warnings and Precautions ( 5.2 )] Alterations in Cardiovascular/Renal Function [see Warnings and Precautions ( 5.3 )] Gastrointestinal Perforation [see Warnings and Precautions ( 5.4 )] Behavioral and Mood Disturbances [see Warnings and Precautions ( 5.5 )] Effects on Bones [see Warnings and Precautions ( 5.6 )] Ophthalmic Effects [see Warnings and Precautions ( 5.7 )] Immunizations [see Warnings and Precautions ( 5.8 )] Serious Skin Rashes [see Warnings and Precautions ( 5.9 )] Effects on Growth and Development [see Warnings and Precautions ( 5.10 )] Myopathy [see Warnings and Precautions ( 5.11 )] Kaposi’s Sarcoma [see Warnings and Precautions ( 5.12 )] Risk of Serious Adverse Reactions in Infants because of Benzyl Alcohol Preservative [see Warnings and Precautions ( 5.13 )] Thromboembolic Events [see Warnings and Precautions ( 5.14 )] Anaphylaxis [see Warnings and Precautions ( 5.15 )] The most common adverse reactions (≥ 10% for EMFLAZA and greater than placebo) are Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, central obesity, and nasopharyngitis ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact PTC Therapeutics, Inc. at 1-866-562-4620 or [email protected] or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

6.1Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. In Study 1 [see Clinical Studies ( 14 )] , the adverse reactions that were associated with deflazacort treatment discontinuation, in decreasing order of frequency, were weight increased, obesity, cataract, and sleep disorder. Most Common Adverse Reactions in Clinical Studies Table 1 lists the adverse reactions that occurred in ≥ 5% of patients in the 0.9 mg/kg/day deflazacort-treated group and that occurred more frequently than in placebo patients in Study 1, which included patients with DMD between the ages of 5 and 15 years.

Table 1: Adverse Reactions that Occurred in ≥ 5% of Deflazacort-Treated Patients and Occurred More Frequently than in Placebo Patients with DMD (Study 1) 1 At 12 weeks placebo patients were re-randomized to receive either deflazacort or an active comparator. Adverse Reaction Deflazacort 0.9 mg/kg/d (N=51) % at 12 weeks Placebo (N=50) % at 12 weeks 1 Cushingoid appearance 33 12 Weight increased 20 6 Increased appetite 14 2 Upper respiratory tract infection 12 10 Cough 12 6 Pollakiuria 12 2 Nasopharyngitis 10 6 Hirsutism 10 2 Central obesity 10 4 Erythema 8 6 Irritability 8 4 Rhinorrhea 8 0 Abdominal discomfort 6 2 Common adverse reactions (≥ 5% of deflazacort-treated patients) that occurred over 52 weeks of exposure to deflazacort 0.9 mg/kg/day in Study 1 and at a higher rate than deflazacort 0.9 mg/kg/day in the 12-week placebo-controlled phase of the trial include Cushingoid appearance (60%), hirsutism (35%), weight increased (28%), erythema (28%), central obesity (25%), abdominal pain/abdominal pain upper (18% combined), pollakiuria (15%), constipation (10%), irritability (10%), abnormal behavior (9%), pyrexia (9%), back pain (7%), rash (7%), contusion (6%), nausea (6%), psychomotor hyperactivity (6%), epistaxis (6%), and skin striae (6%).

Study 1 also evaluated a higher dosage of deflazacort (1.2 mg/kg/day). Compared with the 0.9 mg/kg/day dosage, deflazacort 1.2 mg/kg/day over 52 weeks was associated with a higher incidence of certain adverse reactions, including Cushingoid appearance (69%), erythema (49%), hirsutism (37%), headache (34%), weight increased (32%), constipation (…

🔄 Drug Interactions 209 words ▾

7 DRUG INTERACTIONS Moderate or strong CYP3A4 inhibitors: Give one third of the recommended dosage of EMFLAZA ( 7.1 ) Avoid use of moderate or strong CYP3A4 inducers with EMFLAZA, as they may reduce efficacy ( 7.1 )

7.1CYP3A4 Inhibitors and Inducers Moderate or Strong CYP3A4 Inhibitors The active metabolite of deflazacort, 21-desDFZ, is a substrate of CYP3A4 [see Clinical Pharmacology ( 12.3 )] . Co-administration of deflazacort with clarithromycin, a strong CYP3A4 inhibitor, increased total exposure to 21-desDFZ by about 3-fold. Therefore, give one third the recommended dosage of EMFLAZA when moderate or strong CYP3A4 inhibitors (e.g., clarithromycin, fluconazole, diltiazem, verapamil, grapefruit juice) are used concomitantly with EMFLAZA [see Dosage and Administration ( 2.5 ) and Clinical Pharmacology ( 12.3 )] .

Moderate or Strong CYP3A4 Inducers Co-administration of deflazacort with rifampin, a strong CYP3A4 inducer, significantly decreased the exposure of 21-desDFZ. Avoid concomitant use of strong (e.g., efavirenz) or moderate (e.g., carbamazepine, phenytoin) CYP3A4 inducers with EMFLAZA [see Dosage and Administration ( 2.5 ) and Clinical Pharmacology ( 12.3 )] .

7.2Neuromuscular Blockers Patients receiving corticosteroids, including EMFLAZA, and concomitant therapy with neuromuscular blocking drugs (e.g., pancuronium) may be at increased risk of developing an acute myopathy [see Warnings and Precautions ( 5.11 )] .

👥 Use in Specific Populations ~3 min read ▾

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy Risk Summary Corticosteroids should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Infants born to mothers who have received substantial doses of corticosteroids during pregnancy should be carefully observed for signs of hypoadrenalism. There are no adequate and well-controlled studies with EMFLAZA in pregnant women to inform drug-associated risks.

Corticosteroids, including EMFLAZA, readily cross the placenta. Adverse developmental outcomes, including orofacial clefts (cleft lip, with or without cleft palate) and intrauterine growth restriction, and decreased birth weight, have been reported with maternal use of corticosteroids, including EMFLAZA, during pregnancy. Some epidemiologic studies report an increased risk of orofacial clefts from about 1 per 1000 infants to 3 to 5 per 1000 infants; however, a risk for orofacial clefts has not been observed in all studies.

Intrauterine growth restriction and decreased birth weight appear to be dose-related; however, the underlying maternal condition may also contribute to these risks (see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.

Animal reproduction studies have not been conducted with deflazacort. Animal reproduction studies conducted with other corticosteroids in pregnant mice, rats, hamsters, and rabbits using clinically relevant doses have shown an increased incidence of cleft palate. An increase in embryofetal death, intrauterine growth retardation, and constriction of the ductus arteriosus were observed in some animal species.

Data Human Data Multiple cohort and case-controlled studies in humans suggest that maternal corticosteroid use during the first trimester increases the rate of cleft lip, with or without cleft palate, from about 1/1000 infants to 3-5/1000 infants. Two prospective case-controlled studies showed decreased birth weight in infants exposed to maternal corticosteroids in utero.

8.2Lactation Risk Summary Systemically administered corticosteroids appear in human milk and could suppress growth, interfere with endogenous corticosteroid production, or cause other untoward effects. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for EMFLAZA and any potential adverse effects on the breastfed infant from EMFLAZA. There are no data on the effects on milk production.

8.4Pediatric Use The safety and effectiveness of EMFLAZA for the treatment of DMD have been established in patients 2 years of age and older. Use of EMFLAZA in pediatric patients is supported by a multicenter, randomized, double-blind, placebo- and active-controlled study in 196 males 5 to 15 years of age [see Clinical Studies ( 14 )] . Use of EMFLAZA in patients 2 years to less than 5 years of age is supported by the findings of efficacy and safety in patients 5 years and older with DMD.

Safety and effectiveness in pediatric patients below the age of 2 years have not been established. EMFLAZA Oral Suspension contains benzyl alcohol and is not approved for use in pediatric patients less than 2 years of age. Serious adverse reactions including fatal reactions and “gasping syndrome” occurred in premature neonates and low birth weight infants in the neonatal intensive care unit who received drugs containing benzyl alcohol as a preservative.

In these cases, benzyl alcohol dosages of 99 to 234 mg/kg/day produced high levels of benzyl alcohol and its metabolites in the blood and urine (blood levels of benzyl alcohol were 0.61 to 1.378 mmol/L). Additional adverse reactions included gradual neurological deterioration, seizures, intracranial hemorrhage, hematologic abnormalities, skin breakdown, hepatic and ren…

🤰 Pregnancy ~1 min read ▾

8.1Pregnancy Risk Summary Corticosteroids should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Infants born to mothers who have received substantial doses of corticosteroids during pregnancy should be carefully observed for signs of hypoadrenalism. There are no adequate and well-controlled studies with EMFLAZA in pregnant women to inform drug-associated risks.

Corticosteroids, including EMFLAZA, readily cross the placenta. Adverse developmental outcomes, including orofacial clefts (cleft lip, with or without cleft palate) and intrauterine growth restriction, and decreased birth weight, have been reported with maternal use of corticosteroids, including EMFLAZA, during pregnancy. Some epidemiologic studies report an increased risk of orofacial clefts from about 1 per 1000 infants to 3 to 5 per 1000 infants; however, a risk for orofacial clefts has not been observed in all studies.

Intrauterine growth restriction and decreased birth weight appear to be dose-related; however, the underlying maternal condition may also contribute to these risks (see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.

Animal reproduction studies have not been conducted with deflazacort. Animal reproduction studies conducted with other corticosteroids in pregnant mice, rats, hamsters, and rabbits using clinically relevant doses have shown an increased incidence of cleft palate. An increase in embryofetal death, intrauterine growth retardation, and constriction of the ductus arteriosus were observed in some animal species.

Data Human Data Multiple cohort and case-controlled studies in humans suggest that maternal corticosteroid use during the first trimester increases the rate of cleft lip, with or without cleft palate, from about 1/1000 infants to 3-5/1000 infants. Two prospective case-controlled studies showed decreased birth weight in infants exposed to maternal corticosteroids in utero.

🧒 Pediatric Use ~2 min read ▾

8.4Pediatric Use The safety and effectiveness of EMFLAZA for the treatment of DMD have been established in patients 2 years of age and older. Use of EMFLAZA in pediatric patients is supported by a multicenter, randomized, double-blind, placebo- and active-controlled study in 196 males 5 to 15 years of age [see Clinical Studies ( 14 )] . Use of EMFLAZA in patients 2 years to less than 5 years of age is supported by the findings of efficacy and safety in patients 5 years and older with DMD.

Safety and effectiveness in pediatric patients below the age of 2 years have not been established. EMFLAZA Oral Suspension contains benzyl alcohol and is not approved for use in pediatric patients less than 2 years of age. Serious adverse reactions including fatal reactions and “gasping syndrome” occurred in premature neonates and low birth weight infants in the neonatal intensive care unit who received drugs containing benzyl alcohol as a preservative.

In these cases, benzyl alcohol dosages of 99 to 234 mg/kg/day produced high levels of benzyl alcohol and its metabolites in the blood and urine (blood levels of benzyl alcohol were 0.61 to 1.378 mmol/L). Additional adverse reactions included gradual neurological deterioration, seizures, intracranial hemorrhage, hematologic abnormalities, skin breakdown, hepatic and renal failure, hypotension, bradycardia, and cardiovascular collapse. Preterm, low-birth weight infants may be more likely to develop these reactions because they may be less able to metabolize benzyl alcohol.

When prescribing EMFLAZA Oral Suspension consider the combined daily metabolic load of benzyl alcohol from all sources including EMFLAZA Oral Suspension (EMFLAZA Oral Suspension contains 10.45 mg of benzyl alcohol per mL; EMFLAZA Tablets do not contain benzyl alcohol) and other drugs containing benzyl alcohol. The minimum amount of benzyl alcohol at which serious adverse reactions may occur is not known. At the recommended dose of 0.9 mg/kg/day of EMFLAZA Oral Suspension, patients would receive approximately 0.4 mg/kg/day of benzyl alcohol [see Warnings and Precautions ( 5.13 )] .

Juvenile Animal Toxicity Data Oral administration of deflazacort (0, 0.1, 0.3, and 1.0 mg/kg/day) to juvenile rats from postnatal day (PND) 21 to 80 resulted in decreased body weight gain and adverse effects on skeletal development (including decreased cellularity of growth plate and altered bone distribution) and on lymphoid tissue (decreased cellularity). A no-effect dose was not identified. In addition, neurological and neurobehavioral abnormalities were observed at the mid and/or high dose.

Plasma 21-desDFZ exposure (AUC) at the lowest dose tested (0.1 mg/kg/day) was lower than that in humans at the recommended human dose of EMFLAZA (0.9 mg/kg/day).

🧓 Geriatric Use 21 words ▾

8.5Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with EMFLAZA.

🆘 Overdosage 47 words ▾

10 OVERDOSAGE Treatment of acute overdosage is by immediate gastric lavage or emesis followed by supportive and symptomatic therapy. For chronic overdosage in the face of severe disease requiring continuous steroid therapy, the dosage of EMFLAZA may be reduced temporarily, or alternate day treatment may be introduced.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Deflazacort is a corticosteroid prodrug, whose active metabolite, 21-desDFZ, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown.

12.3Pharmacokinetics Absorption After oral administration in the fasted state, the median T max with deflazacort tablets or suspension is about 1 hour (range 0.25 to 2 hours). Food Effect: Co-administration of deflazacort tablets with a high-fat meal reduced C max by about 30% and delayed T max by one hour, relative to administration under fasting conditions, but there was no effect on the overall systemic absorption as measured by AUC. The bioavailability of deflazacort tablets was similar to that of the oral suspension.

The administration of deflazacort with food or crushed in applesauce did not affect the absorption and bioavailability of deflazacort. Distribution The protein binding of the active metabolite of deflazacort is about 40%. Elimination Metabolism Deflazacort is rapidly converted to the active metabolite 21-desDFZ by esterases after oral administration.

21-desDFZ is further metabolized by CYP3A4 to several other inactive metabolites, including 6β-hydroxy-21-desacetyl deflazacort. Excretion Urinary excretion is the predominant route of deflazacort elimination (about 68% of the dose), and the elimination is almost completed by 24 hours post dose. 21-desDFZ accounts for 18% of the eliminated drug in the urine.

Specific Populations Pediatric Patients The C max values (Geometric mean, %CV) of 21-desDFZ in children (ages 4-11, N=16) and adolescents (ages 12-16, N=8) was 206 ng/mL (95.6%) and 381 ng/mL (37.7%), respectively, on Day 1 after administration of 0.9 mg/kg deflazacort. The AUC inf (Geometric mean, %CV) of 21-desDFZ in children (ages 4-11, N=16) and adolescents (ages 12-16, N=8) was 400 ng•h/mL (87.5%) and 655 ng•h/mL (58.1%) on Day 1 after administration of 0.9 mg/kg deflazacort. Male and Female Patients There are no differences in the pharmacokinetics of 21-desDFZ between males and females.

Racial or Ethnic Groups There are no differences in the pharmacokinetics of 21-desDFZ between Caucasians and non-Caucasians. Patients with Renal Impairment In a study (N=16) comparing subjects with end stage renal disease (creatinine clearance less than 15 mL/min) with healthy matched controls, 21-desDFZ exposure was similar between the groups. Patients with Hepatic Impairment In a study (N=16) comparing subjects with moderate hepatic impairment (Child-Pugh Class B) with healthy matched controls, 21-desDFZ exposure was similar between the groups.

There is no experience in patients with severe hepatic impairment. Drug Interaction Studies In Vivo Assessment of Drug Interactions Compared to administration of deflazacort alone, administration of deflazacort following multiple doses of a strong CYP3A4 and Pgp inhibitor (clarithromycin) resulted in markedly higher C max , AUC last , and AUC inf values of 21-desDFZ. Geometric mean exposure (C max , AUC last , and AUC inf ) of 21-desDFZ ranged from 2.3-fold to 3.4-fold higher following administration of clarithromycin [see Dosage and Administration ( 2.5 )] .

Compared to administration of deflazacort alone, administration of deflazacort following multiple doses of a strong CYP3A4 inducer (rifampicin) resulted in markedly lower C max , AUC last , and AUC inf values of 21-desDFZ. Geometric mean exposures (C max , AUC la st , and AUC inf ) of 21-desDFZ were approximately 95% lower following administration of rifampin [see Drug Interactions ( 7.1 )] . 6β-Hydroxy-21-desacetyl deflazacort, a secondary and inactive metabolite, is not expected to cause any clinically meaningful interactions with the CYP enzymes or transporters.

In Vitro Assessment of Drug Interactions Drug-Metabolizing Enzyme Inhibition 21-desDFZ at clinically relevant concentrations did not…

🧬 Mechanism of Action 40 words ▾

12.1Mechanism of Action Deflazacort is a corticosteroid prodrug, whose active metabolite, 21-desDFZ, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown.

📦 How Supplied / Storage and Handling ~1 min read ▾

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1How Supplied EMFLAZA Tablets 6 mg are white, round with “6” debossed on one side. They are supplied as follows: NDC 52856-501-01 Bottle of 100 tablets 18 mg are white, round with “18” debossed on one side. They are supplied as follows: NDC 52856-502-03 Bottle of 30 tablets 30 mg are white, oval with “30” debossed on one side.

They are supplied as follows: NDC 52856-503-03 Bottle of 30 tablets 36 mg are white, oval with “36” debossed on one side. They are supplied as follows: NDC 52856-504-03 Bottle of 30 tablets EMFLAZA Oral Suspension 22.75 mg/mL is a whitish colored suspension. Supplied as 13 mL in a 30 mL bottle packaged with one bottle adapter and two 1 mL oral dispensers.

NDC 52856-505-22

16.2Storage and Handling Store at 20°C to 25°C (68°F to 77°F). Excursion permitted between 15°C to 30°C (59°F to 86°F). See USP controlled room temperature. Discard any unused EMFLAZA Oral Suspension remaining after 1 month of first opening the bottle.

16.1How Supplied EMFLAZA Tablets 6 mg are white, round with “6” debossed on one side. They are supplied as follows: NDC 52856-501-01 Bottle of 100 tablets 18 mg are white, round with “18” debossed on one side. They are supplied as follows: NDC 52856-502-03 Bottle of 30 tablets 30 mg are white, oval with “30” debossed on one side.

They are supplied as follows: NDC 52856-503-03 Bottle of 30 tablets 36 mg are white, oval with “36” debossed on one side. They are supplied as follows: NDC 52856-504-03 Bottle of 30 tablets EMFLAZA Oral Suspension 22.75 mg/mL is a whitish colored suspension. Supplied as 13 mL in a 30 mL bottle packaged with one bottle adapter and two 1 mL oral dispensers.

NDC 52856-505-22

📦 Storage and Handling 41 words ▾

16.2Storage and Handling Store at 20°C to 25°C (68°F to 77°F). Excursion permitted between 15°C to 30°C (59°F to 86°F). See USP controlled room temperature. Discard any unused EMFLAZA Oral Suspension remaining after 1 month of first opening the bottle.

📋 Description 183 words ▾

11 DESCRIPTION The active ingredient in EMFLAZA is deflazacort (a corticosteroid). Corticosteroids are adrenocortical steroids, both naturally occurring and synthetic. The molecular formula for deflazacort is C 25 H 31 NO 6 .

The chemical name for deflazacort is (11β,16β)-21-(acetyloxy)-11-hydroxy-2'-methyl-5'H-pregna-1,4-dieno[17,16-d]oxazole-3,20-dione, and the structure is: Deflazacort is a white to off white, odorless fine powder and has a molecular weight of 441.517. Deflazacort is freely soluble in acetic acid and dichloromethane and soluble in methanol and acetone. EMFLAZA for oral administration is available as an immediate-release tablet in strengths of 6, 18, 30 and 36 mg and an immediate-release oral suspension in a strength of 22.75 mg/mL.

Each tablet contains deflazacort and the following inactive ingredients: colloidal silicon dioxide, lactose monohydrate, magnesium stearate, and pre-gelatinized corn starch. The oral suspension contains deflazacort and the following inactive ingredients: acetic acid, aluminum magnesium silicate, benzyl alcohol, carboxymethylcellulose sodium, polysorbate 80, purified water, and sorbitol. The structural formula for EMFLAZA is deflazacort (a corticosteroid).

Corticosteroids are adrenocortical steroids, both naturally occurring and synthetic. The molecular formula for deflazacort is C25H31NO6. The chemical name for deflazacort is (11β,16β)-21-(acetyloxy)-11-hydroxy-2'-methyl-5'H-pregna-1,4-dieno[17,16-d]oxazole-3,20-dione.

💬 Information for Patients ~3 min read ▾

17 PATIENT COUNSELING INFORMATION Advise the patients and/or caregivers to read the FDA-approved patient labeling if EMFLAZA Oral Suspension is prescribed ( Instructions for Use ). Administration Warn patients and/or caregivers to not stop taking EMFLAZA abruptly or without first checking with their healthcare providers as there may be a need for gradual dose reduction to decrease the risk of adrenal insufficiency [see Dosage and Administration ( 2.3 ) and Warnings and Precautions ( 5.1 )] . EMFLAZA may be taken with or without food.

Do not take EMFLAZA with grapefruit juice. Tablets EMFLAZA Tablets may be taken whole or crushed and taken immediately after mixing with applesauce. Oral Suspension EMFLAZA Oral Suspension must be shaken well prior to measuring out each dose with the enclosed oral dispenser.

The EMFLAZA Oral Suspension dose may be placed in 3-4 ounces of juice (except grapefruit juice) or milk, mixed thoroughly, and immediately administered. Discard any unused EMFLAZA Oral Suspension remaining after 1 month of first opening the bottle. Increased Risk of Infection Tell patients and/or caregivers to inform their healthcare provider if the patient has had recent or ongoing infections or if they have recently received a vaccine.

Medical advice should be sought immediately if the patient develops fever or other signs of infection. Patients and/or caregivers should be made aware that some infections can potentially be severe and fatal. Warn patients who are on corticosteroids to avoid exposure to chickenpox or measles and to alert their healthcare provider immediately if they are exposed [see Warnings and Precautions ( 5.2 )] .

Alterations in Cardiovascular/Renal Function Inform patients and/or caregivers that EMFLAZA can cause an increase in blood pressure and water retention. If this occurs, dietary salt restriction and potassium supplementation may be needed [see Warnings and Precautions ( 5.3 )] . Behavioral and Mood Disturbances Advise patients and/or caregivers about the potential for severe behavioral and mood changes with EMFLAZA and encourage them to seek medical attention if psychiatric symptoms develop [see Warnings and Precautions ( 5.5 )] .

Decreases in Bone Mineral Density Advise patients and/or caregivers about the risk of osteoporosis with prolonged use of EMFLAZA, which can predispose the patient to vertebral and long bone fractures [see Warnings and Precautions ( 5.6 )] . Ophthalmic Effects Inform patients and/or caregivers that EMFLAZA may cause cataracts or glaucoma and advise monitoring if corticosteroid therapy is continued for more than 6 weeks [see Warnings and Precautions ( 5.7 )] . Vaccination Advise patients and/or caregivers to bring immunizations up-to-date according to immunization guidelines prior to starting therapy with EMFLAZA.

Live-attenuated or live vaccines should be administered at least 4 to 6 weeks prior to starting EMFLAZA. Inform patients and/or caregivers that they may receive concurrent vaccinations with use of EMFLAZA, except for live-attenuated or live vaccines. [see Warnings and Precautions ( 5.8 )] . Serious Skin Rashes Instruct patients and/or caregivers to seek medical attention at the first sign of a rash [see Warnings and Precautions ( 5.9 )] .

Drug Interactions Certain medications can cause an interaction with EMFLAZA. Advise patients and/or caregivers to inform their healthcare provider of all the medicines the patient is taking, including over-the-counter medicines (such as insulin, aspirin or other NSAIDS), dietary supplements, and herbal products. Inform patients and/or caregivers that alternate therapy, dosage adjustment, and/or special test(s) may be needed during the treatment.

PTC Therapeutics Manufactured for: PTC Therapeutics, Inc. Warren, NJ 07059 U.S.A. EMFLAZA Oral Suspension made in Spain EMFLAZA ® is a trademark of PTC Therapeutics, Inc.

Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗
← 52856-0503-03 · PTC 52856-0505-22 · PTC →
For educational and professional reference only — not medical advice. Pricing reflects published NADAC and CMS ASP (free public data) and may differ from your acquisition cost; always verify before billing or dispensing.