Important Safety Information
Prescribing Information

Help boys with DMD preserve their "here and now"

As the first and only FDA‑approved corticosteroid for boys with DMD as young as age 2, EMFLAZA is designed to help maintain muscle strength and function.

In phase 1 (from baseline to 12 weeks) of a 52‑week clinical trial with 196 boys aged 5 to 15 years with DMD, the effectiveness and safety of EMFLAZA were compared to placebo (sugar pill). After 12 weeks of treatment, patients taking EMFLAZA had significantly improved muscle strength compared with placebo (0.15 change in strength score vs -0.10 change in strength score).

Make the Most
of His Now

Help delay dmd progression,
When it matters

In a 10-year follow-up study measuring 3 important DMD events, deflazacort showed differences over time compared to prednisone by helping boys maintain:

In a study of 440 boys aged 2 to 28 years with DMD, the effectiveness and safety of deflazacort were compared with prednisone.

Age at loss of ambulation with deflazacort vs prednisone

Age at Loss of ambulation

Bello 2015 Study Summary

Prednisone/prednisolone and deflazacort regimens in CINRG Duchenne Natural History Study

Objective: This study observed the age at loss of independent ambulation (LoA) and side-effect profiles associated with different glucocorticoid corticosteroid (GC) regimens in DMD.

Methods: This observational study assessed 340 participants aged 2 to 28 years in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS). LoA was defined as continuous wheelchair use. Effects of prednisone or prednisolone/deflazacort, administration frequency, and dose were analyzed by time-varying Cox regression (a method to estimate survival time). Side-effect frequencies were compared using x2 test.

Results: Participants treated 1 year or longer while ambulatory showed a 3-year median delay in LoA. Fourteen different regimens were observed. Non-daily treatment was common for prednisone (37%) and rare for deflazacort (3%). Deflazacort was associated with later LoA than prednisone. Average dose was lower for daily prednisone (75% of the recommended dose) than daily deflazacort (83% of the recommended dose). Deflazacort showed higher frequencies of growth delay, cushingoid appearance, and cataracts, but not weight gain.

Please note
This study is not in the approved prescribing label for EMFLAZA, but is consistent with the information that is included. Please talk to your son's healthcare provider if you have any questions.

Help support him
as he grows

Once your son starts taking deflazacort, it's important for him to stay on treatment as it was shown to preserve muscle function in more places than just his arms and legs.

In a 13-year study, scoliosis was delayed and lung function was preserved for longer in boys taking deflazacort.

A 13-year study of 435 boys with DMD assessed patients' ages simultaneously with DMD progression events, including scoliosis onset and decrease in lung function.

Here & now with
the Kasners

Sarah and Dan work together to care for their two sons with DMD. Watch how they try to have fun and enjoy life as much as they can.

View Video Transcript
Emflaza Safety Profile

Indication & Important Safety Information for EMFLAZA® (deflazacort)

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.

Indication & Important Safety Information for EMFLAZA® (deflazacort)

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.

Prescription Start Form

Indication & Important Safety Information for EMFLAZA® (deflazacort)

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.