Learn how to talk to your son's healthcare provider about staying on EMFLAZA.



Voiceover: Welcome to an informative presentation from two real DMD specialists, Dr. Saunders M. Bernes and Dr. Michelle Figueroa. They're actual neurology experts and they're here to help us understand some of the science behind EMFLAZA, right here and right now.

The Science Behind EMFLAZA® (deflazacort)

Voiceover: Dr. Bernes, you know all about EMFLAZA, also known as deflazacort. Can you tell us a little bit about the efficacy and safety?

Muscle strength after 3 months

Dr. Bernes: I believe it's important to talk about the primary outcome in the pivotal trial that led to the FDA approval. This data is from Dr. Robert Griggs and a team of researchers who evaluated 196 patients age 5 to 15 years of age with Duchenne muscular dystrophy.

Dr. Bernes: The team investigated the efficacy and the safety of EMFLAZA compared with placebo over three months. After the three months of treatment, EMFLAZA improved muscle strength compared to placebo.

Improved muscle strength after 3 months vs placebo

Dr. Bernes: The safety of EMFLAZA was evaluated over the year and this was a group of boys who had weakness less than five years of age. After 12 weeks of treatment, EMFLAZA at a dose of 0.9 milligrams per kilogram improved muscle strength through a MRC evaluation of 0.15 versus placebo with loss of function of 0.10.

Voiceover: Thank you Dr. Bernes for telling us about muscle strength and EMFLAZA.

EMFLAZA® (deflazacort) OVER TIME

Voiceover: Understanding how a treatment works over the long-term can be very important. Dr. Figueroa, can you show us how EMFLAZA has worked over long periods of time?

Dr. Figueroa: So the study, it was done in over 20 different centers in nine different countries and in patients with Duchenne muscular dystrophy, looking at how it was progressing in patients who were aged 2- to 28-years old.

Dr. Figueroa: The limitations were noted, they didn't do anything to change anything, so steroid use wasn't randomized. The comparisons between deflazacort and prednisone have not been included in the approved prescribing information because prednisone is not an approved treatment for Duchenne muscular dystrophy by the FDA.

Dr. Figueroa: And the analyses that included the comparison of multiple corticosteroid agents noted that comparison of several of them as well as different regimens, such as daily dosing versus intermittent dosing of different corticosteroids. And an in-depth analysis of specific dosing schedules and actual doses themselves wasn't possible in the long-term observational study. And potential differences were noted in different socioeconomic status of patients as well as their caregivers. And deflazacort at that time was not commercially available in the United States.

Followed 9 different key milestones

Dr. Figueroa: And through this study what we were able to say is that they followed nine different key milestones for those boys.

Help delay dmd progression, when it matters most

Dr. Figueroa: And the three most important ones that were gleaned from that was coming from lying on his back to standing up and the time that that that took, how long the boys were able to walk without any assistance, their ability to maintain their hand-to-mouth function, as well as lung function over time.

There were functional benefits with deflazacort

Dr. Figueroa: And what we were able to note is that there were functional benefits in this long-term real-world data. And what we were able to note is that deflazacort tended to allow boys to stand much more quickly and for a much longer period of time than in comparison to prednisone or no steroids. Boys were walking on average about 2.7 years longer with deflazacort and that was the ability to walk without any assistance. And they were able to maintain their hand-to-mouth function for at least about an average of 2.7 years longer with deflazacort.

Voiceover: Thank you for that engaging explanation of EMFLAZA over the years, Dr. Figueroa.

Age at loss of ambulation

Voiceover: The relationship between treatment and age at loss of ambulation is important to understand. Dr. Bernes, can you explain the real-world data for EMFLAZA and ambulation?

Dr. Bernes: The first study that I would like to talk about is an observational study published by Bello and colleagues in 2015. This is a study that included over 340 patients who were at 2 to 28 years of age, were enrolled in the CINRG Duchenne Natural History study, and evaluated for loss of ambulation. They also evaluated for side effect profiles associated with different corticosteroid regimens.

Age at loss of ambulation

Dr. Bernes: Loss of ambulation in this study was defined as wheelchair use or the inability to walk 10 minutes unaided.

Dr. Bernes: The efficacy of both deflazacort and prednisone can be seen in this graph, which is a Kaplan Meier curve. It shows the loss of ambulation for patients taking deflazacort versus those taking prednisone.

Daily deflazacort delayed the loss of ambulation by 2.7 years

Dr. Bernes: The study looked at outcome for loss of ambulation, which showed that the real-world study of over 435 patients with Duchenne muscular dystrophy that daily deflazacort delayed the loss of ambulation by 2.7 years compared to a daily dose of prednisone.

Voiceover: That was very helpful, Dr. Bernes. Glad you could help us take a deeper look into that data.

Supporting more than just arms and legs

Voiceover: EMFLAZA may help more than just muscle strength. Dr. Figueroa, can you teach us about the effects on lung function and the need for scoliosis surgery?

Helping boys as they grow

Dr. Figueroa: That study that followed boys over about 11 years was the Cincinnati hospital. In its study design was that it had 430 boys that it followed over the course of about 11 to 13 years. And what was noted is that there were also benefits from deflazacort in regard to scoliosis as well as lung function.

Delayed onset of scoliosis

Dr. Figueroa: And what we were able to see was that use of deflazacort itself delayed the onset of scoliosis and lowered that risk as well as delayed any time that was needed for any corrective measures or surgeries for scoliosis itself.

Preserved lung function

Dr. Figueroa: Also what was noted was that it did preserve lung function in boys for a longer period of time. This was measured by what we call forced vital capacity, so it's a objective measure, and what we were noted that was almost a 10% higher forced vital capacity in boys who were using deflazacort versus prednisone.

Voiceover: Very interesting, Dr. Figueroa. That is some compelling data. And now, we'd like to go over the EMFLAZA Important Safety Information.


What is EMFLAZA® (deflazacort) used for?

EMFLAZA is a prescription medicine used to treat Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

When should I not take EMFLAZA?

Do not use if you have had hypersensitivity, including allergic reactions, to deflazacort or any of the inactive ingredients.

What warnings should I know about EMFLAZA?

  • EMFLAZA can cause changes in endocrine function. Do not stop taking EMFLAZA, or change the amount you are taking, without first checking with your healthcare provider, as there may be a need for gradual dose reduction to decrease the risk of adrenal insufficiency and steroid “withdrawal syndrome”. Acute adrenal insufficiency can occur if corticosteroids are withdrawn abruptly, and can be fatal. A steroid “withdrawal syndrome,” seemingly unrelated to adrenocortical insufficiency, may also occur following abrupt discontinuance of corticosteroids. For patients already taking corticosteroids during times of stress, the dosage may need to be increased.
  • There is an increased risk of infection when taking EMFLAZA. Tell the 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.
  • EMFLAZA can cause an increase in blood pressure and water retention. If this occurs, dietary salt restriction and potassium supplementation may be needed.
  • There is an increased risk of developing a hole in the stomach or intestines in patients with certain stomach or intestine disorders when taking corticosteroids like EMFLAZA.
  • EMFLAZA can cause severe behavioral and mood changes. Seek medical attention from the health care provider if any behavioral or mood changes develop.
  • There is a risk of osteoporosis with prolonged use of EMFLAZA, which can lead to vertebral and long bone fractures.
  • EMFLAZA may cause cataracts or glaucoma and a health care provider should monitor for these conditions if corticosteroid therapy is continued for more than 6 weeks.
  • Immunizations should be 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. Live-attenuated or live vaccines should not be used in patients taking EMFLAZA.
  • EMFLAZA can cause serious skin rashes. Seek medical attention at the first sign of a rash.
  • Rare instances of anaphylaxis have occurred in patients receiving corticosteroid therapy, including EMFLAZA.

What should I tell my health care provider?

Tell the health care provider about all medical conditions, including if the patient:

  • is pregnant or planning to become pregnant. EMFLAZA® (deflazacort) can harm your unborn baby.
  • is breastfeeding or planning to breastfeed. EMFLAZA may appear in breastmilk and could affect a nursing child.

Certain medications can cause an interaction with EMFLAZA. Tell your healthcare provider of all the medicines you are taking, including over-the-counter medicines (such as insulin, aspirin or other NSAIDS), dietary supplements, and herbal products. Alternate treatment, dosage adjustment, and/or special test(s) may be needed during the treatment.

What are the side effects of EMFLAZA?

The most common side effects of EMFLAZA include facial puffiness or Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, frequent daytime urination, unwanted hair growth, central obesity, and colds. These are not all of the possible side effects of EMFLAZA. Call your doctor for medical advice about side effects.

To report an adverse event, please call 1-866-562-4620 or email at usmedinfo@ptcbio.com. You may also report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch.

Emflaza® is a registered trademark of PTC Therapeutics, Inc.
© 2023 PTC Therapeutics, Inc. All Rights Reserved.
US-EMF-0411 06/23