Specialty Pharmacy Services
Deflazacort (Emflaza)
Oral Tablet or Suspension · Once Daily
Deflazacort (Emflaza) is the only FDA-approved corticosteroid specifically indicated for Duchenne muscular dystrophy. Accord Specialty Pharmacy provides deflazacort with prior authorization support, PTC Cares coordination, and doorstep delivery.
Drug at a Glance
- Drug Class Corticosteroid (Glucocorticoid)
- Generic Name Deflazacort
- Brand Name Emflaza®
- Route Oral, once daily (with or without food)
- Manufacturer PTC Therapeutics (brand Emflaza) / Multiple generics
- FDA Approved 2017
Clinically Reviewed By · Accord Clinical Team, PharmD, RPh
Deflazacort (Emflaza) at a Glance
| Feature | Deflazacort (Emflaza) |
|---|---|
| Generic Name | Deflazacort |
| Brand Name | Emflaza® |
| Drug Class | Corticosteroid |
| FDA Approved For | Duchenne Muscular Dystrophy (DMD) |
| Approved Age | 2+ years (brand Emflaza, generic suspension) · 5+ years (generic tablets) |
| Dosage Forms | Tablets, Oral Suspension |
| Available Generic | Yes |
| Specialty Pharmacy Required | Often required by insurance |
| Prior Authorization | Frequently required |
| Copay Assistance Available | Yes, for eligible commercially insured patients |
| Home Delivery | Available through Accord Specialty Pharmacy |
FDA-Approved Use
What Deflazacort (Emflaza) Is Used For
Duchenne Muscular Dystrophy
DMD — PATIENTS 2 YEARS AND OLDERDMD is a rare, progressive genetic disease caused by mutations in the dystrophin gene. Without functioning dystrophin, muscle cells are damaged with normal activity and progressively replaced by scar tissue. Deflazacort is the only FDA-approved corticosteroid specifically indicated for DMD and has been shown to slow muscle strength decline and extend the period of independent walking.1,2
- FDA-approved for patients 2 years of age and older (brand Emflaza and generic oral suspension)
- Generic deflazacort tablets are approved for patients 5 years and older
- Dose is calculated based on your child's weight and adjusted as they grow
Your child's neurologist and care team are the right people to discuss how deflazacort fits into the overall DMD management plan. Learn more about Duchenne muscular dystrophy from the Muscular Dystrophy Association ↗, or read about Rare and genetic disease pharmacy services →
What Is Deflazacort (Emflaza)?
Deflazacort (Emflaza) is the first and only FDA-approved corticosteroid specifically indicated for Duchenne muscular dystrophy, and it is one of the two corticosteroids used as standard of care for boys living with this condition, alongside prednisone. Clinical trial data has shown that it can slow the decline of muscle strength and prolong the ability to walk.1,2,4
Deflazacort works through anti-inflammatory and immunosuppressive effects. The precise way it provides benefit in DMD is not fully understood, but corticosteroid therapy is recognized as one of the most important interventions for slowing disease progression in Duchenne muscular dystrophy.2 Dosing is weight-based, which means your child's neurologist will calculate the right dose for your child's current weight and adjust it as they grow.
Deflazacort is available as a tablet or oral suspension and can be taken with or without food. Because it is a specialty medication, Accord coordinates every step of the process between the prescriber, your insurance plan, and PTC Cares, the manufacturer's patient support program. Learn how Accord supports families →
Deflazacort Side Effects and What to Watch For
Like all corticosteroids, deflazacort can cause side effects. Your child's care team will discuss what to expect before starting treatment, and monitoring is a standard and ongoing part of DMD management.
One critical point for caregivers: deflazacort must never be stopped abruptly. If your child has been taking deflazacort for more than a few days, the dose must be tapered gradually under the guidance of the prescriber. Stopping suddenly can cause adrenal insufficiency, which can be serious. Always consult your child's care team before making any changes to the medication schedule.2 If anything concerns you, call Accord's pharmacy team at 1-855-222-6730.
Corticosteroid-Related Effects2
- Facial and truncal fat redistribution, sometimes called a Cushingoid or "moon face" appearance
- Weight gain and increased appetite
- Unwanted hair growth (hirsutism)
- Upper respiratory infections, colds, and cough
- Increased urinary frequency
Endocrine and Mood-Related Effects2
- Deflazacort can raise blood sugar and may unmask or worsen diabetes; regular blood glucose monitoring is recommended, especially with long-term use
- Mood and behavioral changes can occur during treatment, and in some cases depressive symptoms have been reported after stopping treatment — contact your child's care team if you notice these changes
- Avoid grapefruit juice, which can affect how deflazacort is processed by the body
Infection Risk and Vaccinations2
- Deflazacort suppresses the immune system, increasing the risk of infections including respiratory infections
- If your child is exposed to chickenpox or measles while on deflazacort and has not been vaccinated, contact your child's care team right away
- Live vaccines should be completed at least 4 to 6 weeks before starting deflazacort — discuss the vaccination schedule with your child's care team before beginning treatment
- Report any signs of fever, unusual infection, or illness promptly
Do Not Stop Abruptly2
- Deflazacort must never be stopped suddenly without guidance from your child's prescriber — abrupt discontinuation can cause adrenal insufficiency, a potentially serious condition
- If your child has been taking deflazacort for more than a few days, the dose must be tapered gradually
- Symptoms of adrenal insufficiency include unusual weakness, dizziness, nausea, and low blood pressure — seek medical attention if these occur after a missed or reduced dose
- Always consult your child's care team before making any changes to the medication schedule
Deflazacort vs. Prednisone for DMD
Deflazacort and prednisone are both corticosteroids used in Duchenne muscular dystrophy, and families often want to understand how they differ. Your child's neurologist is the right person to decide which is appropriate for your child. The comparisons below are drawn from the largest head-to-head trial of the two drugs in DMD to date.4
| Deflazacort (Emflaza) | Prednisone | |
|---|---|---|
| FDA-Approved for DMD2 | Yes, the only corticosteroid with an FDA indication specifically for DMD | No, used off-label for DMD |
| Typical Weight Gain4 | Less weight gain than prednisone in a head-to-head trial | Significantly more weight gain than deflazacort in the same trial |
| Effect on Growth (Height)4 | Greater decrease in growth percentile than prednisone in the same trial | Less growth suppression than deflazacort in the same trial |
| Cushingoid Appearance4 | Occurred less frequently than with prednisone in the trial | Occurred more frequently than with deflazacort in the trial |
| Cataracts4 | Higher rate than prednisone in the trial (dose-dependent) | Lower rate than deflazacort in the trial |
| Cost and Assistance3 | Higher list price, manufacturer copay assistance available through PTC Cares | Lower cost, widely available generic |
| Insurance Step Therapy | Some plans require a documented prednisone trial first | Often the required first-line step therapy option |
These comparisons are based on published clinical studies. The choice between medications should be individualized by your child's treating specialist.
Some insurance plans require a documented trial of prednisone before approving deflazacort. Our team can help navigate this step therapy requirement if it applies to your plan. Call 1-855-222-6730 with any questions.
Prior Authorization for Deflazacort (Emflaza)
Most insurance plans require prior authorization before covering deflazacort. For deflazacort, this typically includes genetic confirmation of DMD, documentation of elevated creatinine kinase levels, and confirmation that your child meets the age requirement. Some plans also require a trial of prednisone before they will approve deflazacort — a process called step therapy. If your child's plan has this requirement, our team works alongside the prescriber to document the clinical basis for deflazacort specifically.
PTC Cares, the manufacturer's patient support program, also provides case managers who are experienced in navigating insurance processes for families. Our team coordinates with PTC Cares on your behalf when appropriate. If your plan issues a denial, we work with your child's prescriber to submit a formal appeal. Information for prescribers →
Prior Authorization Process
Documentation
We request genetic confirmation of DMD, creatinine kinase levels, and diagnosis records from your child's prescriber
PA Submission
Prior authorization submitted to your insurance plan with full clinical documentation
Plan Decision
Approval issued, or denial received — step therapy requirements addressed if applicable
Appeal if Denied
We build a formal appeal with your child's prescriber to support your case
Note on appeals: Many initially denied requests — including those involving step therapy requirements — are approved on formal appeal when documentation is complete and well-organized. Our team manages this process alongside your child's prescriber.
Deflazacort Cost and Emflaza Financial Assistance
Deflazacort cost and Emflaza cost can be a significant concern for families. PTC Therapeutics offers a patient support program called PTC Cares, with case managers who help families navigate insurance, copay assistance, and patient assistance. Many families pay less than the list price after enrolling in these programs.
Commercially Insured
PTC Cares Copay Assistance Program
$0 as low as, for eligible commercially insured patients
PTC Cares provides dedicated case managers who work with families to apply copay assistance at the pharmacy. For enrollment and information, call 1-844-EMFLAZA (1-844-363-5292) or visit emflaza.com. Not available for patients with Medicare, Medicaid, or other government insurance.3
Bridge — During Insurance Approval Wait
PTC Cares Bridge Program
$0 no cost during the insurance approval waiting period
If your child has just been prescribed deflazacort and is waiting for insurance approval, the PTC Cares Bridge Program provides Emflaza at no cost during the waiting period so treatment does not need to be delayed. Call 1-844-EMFLAZA to ask about eligibility.3
Uninsured
PTC Cares Patient Assistance
$0 for eligible uninsured patients
If your child does not have insurance coverage and meets eligibility criteria, PTC Cares may provide brand Emflaza free of charge. A PTC Cares case manager will guide your family through the application process. Call 1-844-EMFLAZA (1-844-363-5292) to discuss eligibility.3
Medicare or Government Insurance
Independent Foundation Grants
Varies by foundation and funding availability
Manufacturer copay programs are not available for patients with Medicare, Medicaid, or other government-funded insurance. PTC Cares case managers can refer families to independent foundations that may provide financial assistance. Our team can also help identify available options.3
Have questions about your family's specific coverage? Call 1-855-222-6730 or contact us online. Our team reviews your coverage and helps connect you to every program your family may qualify for.
Getting Started
What to Expect with Accord
Referral Sent
Your child's prescriber sends a referral to Accord by fax or online referral form.
Insurance Verified
We confirm your coverage, benefits, and any cost-sharing or step-therapy requirements.
Prior Authorization
We submit clinical documentation and manage the PA process — including any step therapy response — on your behalf.
Financial Assistance
We connect your family with PTC Cares and other programs that may reduce out-of-pocket cost.
Doorstep Delivery
Medication ships directly to your door in states where Accord is licensed.
Have Questions About Deflazacort?
Common Questions About Deflazacort (Emflaza)
Does Accord Specialty Pharmacy carry deflazacort (Emflaza)?
Yes. Accord is a licensed specialty pharmacy that dispenses both brand Emflaza and generic deflazacort. We manage prior authorization, copay assistance coordination, and doorstep delivery in states where Accord holds an active pharmacy license.
How do I get my child's deflazacort prescription started at Accord?
Your child's prescriber can send a referral directly to Accord using our referral form, or by faxing to 386-456-3000. You can also call us at 1-855-222-6730 and our team will walk you through the process. A PTC Cares case manager can also be involved from the prescriber's side.
Is there a generic version of Emflaza?
Yes. Aurobindo Pharma makes a generic deflazacort tablet, and Cranbury Pharmaceuticals (Tris Pharma) makes a generic deflazacort oral suspension. There is one important distinction: the generic tablet is approved for patients 5 years of age and older, while brand Emflaza and the generic oral suspension are both approved starting at 2 years of age.2 For children under 5 who need a tablet, brand Emflaza may be the appropriate option; the generic oral suspension is another option regardless of age. If your child's prescriber writes "Dispense as Written" (DAW) on the prescription, brand Emflaza will be dispensed. Our team can help you understand what applies to your child's situation.
Is deflazacort a steroid?
Yes. Deflazacort is a corticosteroid, a class of medications that includes prednisone and prednisolone. Corticosteroids reduce inflammation and suppress the immune system, which is how they provide benefit in DMD. Deflazacort is not an anabolic steroid, which is a different class of drug associated with performance enhancement.2 If you have questions about what being on a corticosteroid means for your child's day-to-day care, our pharmacy team can be reached at 1-855-222-6730.
How does deflazacort compare to prednisone for DMD?
Both are corticosteroids used in DMD management, but deflazacort is the only one FDA-approved specifically for this indication in the United States, while prednisone is used off-label.2 See our full deflazacort vs. prednisone comparison for a side-by-side look at weight gain, growth effects, cost, and insurance step therapy. The choice between them is a clinical decision your child's neurologist is best positioned to make.
Why is the dose different from what other families report?
Deflazacort is dosed based on your child's weight, which means every child's dose will be different. As your child grows, the dose will be adjusted accordingly.2 Your child's prescriber will calculate the right dose and guide any changes over time. If you have questions about whether the dose seems right, your child's neurologist and our pharmacists are the best resources.
What if my insurance denies deflazacort or requires prednisone first?
Step therapy requirements — where insurers require a trial of prednisone before approving deflazacort — do occur. Our team works alongside your child's prescriber to document the clinical rationale for deflazacort and submit a prior authorization or appeal. PTC Cares case managers are also experienced in supporting families through this process. Many denials are overturned on appeal when documentation is thorough. Call us at 1-855-222-6730 and we will get started right away.
Can Emflaza (deflazacort) tablets be crushed?
Yes. According to the prescribing information, Emflaza tablets can be given whole or crushed and mixed with applesauce, and should be taken right after mixing.2 The oral suspension formulation is also available and can be measured to the exact prescribed dose if that works better for your child. Call us at 1-855-222-6730 if you have questions about the right formulation for your child.
What should I do if my child misses a dose of deflazacort?
Give the missed dose as soon as you remember, unless it is almost time for the next dose, in which case skip it and resume the regular schedule. Do not give a double dose to make up for a missed one. Because deflazacort should never be stopped abruptly, missing several doses in a row is different from missing a single dose. If your child misses more than one dose in a row, or you are unsure what to do, contact your child's prescriber or call our pharmacists at 1-855-222-6730 right away.
Can my child receive vaccines while taking Emflaza (deflazacort)?
Deflazacort suppresses the immune system, so live vaccines should generally be completed at least 4 to 6 weeks before starting treatment rather than during it.2 Non-live (inactivated) vaccines are generally considered compatible with ongoing treatment, but your child's pediatrician and neurologist should confirm the vaccination schedule together. Always check with your child's care team before any vaccination while your child is on deflazacort.
Does Emflaza (deflazacort) affect growth?
Like other corticosteroids, deflazacort can affect a child's growth rate, and your child's care team will monitor height and growth regularly throughout treatment.2 In the largest head-to-head trial of deflazacort and prednisone in DMD, deflazacort was associated with somewhat greater growth suppression than prednisone, though it also caused less weight gain.4 See our deflazacort vs. prednisone comparison for more detail. Report any concerns about your child's growth to their neurologist.
Does Emflaza (deflazacort) cause weight gain?
Weight gain and increased appetite are among the most commonly reported effects of deflazacort, as with other corticosteroids.2 In a large head-to-head trial, deflazacort was associated with significantly less weight gain than prednisone.4 Your child's care team will monitor weight throughout treatment as part of routine DMD management. See our deflazacort vs. prednisone comparison for more detail.
For Families
Ready to Get Started?
Our team coordinates everything — insurance verification, prior authorization, PTC Cares enrollment, and delivery. Call us or ask your child's prescriber to send a referral, and we take it from there.
Call 1-855-222-6730For Prescribers
Refer a Patient to Accord
Download the deflazacort referral form, complete all sections, and fax to 386-456-3000. We confirm receipt within one business day and manage prior authorization on your behalf.
Download Referral FormReferences (4)
- Muscular Dystrophy Association. Duchenne Muscular Dystrophy (DMD). mda.org
- Emflaza (deflazacort) Prescribing Information. PTC Therapeutics, Inc. DailyMed. dailymed.nlm.nih.gov
- PTC Cares. Patient Support Programs for Emflaza (deflazacort). PTC Therapeutics. emflaza.com
- Griggs RC, Miller JP, Greenberg CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016;87(20):2123–2131. pubmed.ncbi.nlm.nih.gov