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March 16, 2017

March 16, 2017: An Open Letter to the Duchenne Community

To All Members of the Duchenne Community:

Today marks an important milestone in the effort to bring EMFLAZATM (deflazacort) to patients age five years and older in the Duchenne Muscular Dystrophy community. We are pleased to announce that we have reached an agreement for PTC Therapeutics, Inc. to acquire EMFLAZA, including the full commercialization rights. PTC is really the ideal partner to advance EMFLAZA. We believe this transaction ensures that thousands more children with Duchenne in the United States will have wide and consistent access to this important therapy. You can view the press release issued by PTC earlier today here.

We are proud of the success we achieved in securing FDA approval of EMFLAZA – the first time this drug has been approved for Duchenne muscular dystrophy anywhere in the world. Without FDA approval and the research needed for approval, there was not a complete understanding of the drug, no private or public insurance programs would cover EMFLAZA and very few health care providers would prescribe a non-FDA approved drug. More than 90 percent of the U.S. Duchenne population did not have access to this medicine. Of the 15,000 Duchenne patients in the U.S., 9,000 are age five or older. Now that EMFLAZA is FDA-approved in the U.S., a greater number of patients will have access to this important medicine. It is our great hope that this is a pathway for the development of additional treatments and more advanced care for the Duchenne community and that Marathon’s work will help DMD patients for generations to come.

We believe this transaction will help create the opportunities needed to ensure the greatest number of patients with Duchenne who might benefit from EMFLAZA can receive this drug. To guide us in our decision-making over the past several weeks, we conducted a comprehensive review of all the alternatives available to us. We had numerous discussions with advocacy groups, doctors, healthcare economists, payors, elected officials as well as others. We thank all of you for your valuable input and feedback.

Following this period of review, we concluded that the best path forward is for PTC to commercialize EMFLAZA and to bring it to the DMD community. PTC has deep-rooted experience with Duchenne and will build upon the progress Marathon has made to date in securing FDA approval for EMFLAZA and bringing it to the point of commercialization. PTC provides a broader platform for patient access and is committed to ongoing research to advance the knowledge, diagnosis and treatment of this rare and fatal genetic disorder.

Our commitment to this community is strong. All of us at Marathon have been deeply touched by so many of you, and I want you to know how much we appreciate your commitment to finding the best possible ways to manage the effects of this disease. Every day, I am humbled as I learn of the challenges that all of you face, and it is a great honor to have been able to work with you to improve the lives of patients and by extension their families.

Finally, I want to thank all of the Marathon employees who have been the engine of our progress. Their hard work and commitment to addressing the unmet needs of the Duchenne community have been inspiring. PTC is truly fortunate to be adding many of these talented and passionate individuals to the combined team.

Thank you for your support and commitment. We look forward to continuing to speak with so many of you now and in the future.


Jeff and the Marathon Team

ABOUT EMFLAZATM (deflazacort)
EMFLAZA is indicated for the treatment of Duchenne muscular dystrophy in patients 5 years of age and older.

EMFLAZA is not for patients who are allergic to deflazacort or any of the inactive ingredients in EMFLAZA.

Patients should not stop taking EMFLAZA, or change the amount they are taking, without first checking with their healthcare provider, as there may be a need for gradual dose reduction to decrease the risk of serious side effects.
Corticosteroids, such as EMFLAZA can cause:

  • Hyperglycemia, altered glucose metabolism
  • Increased risk of infection
  • Changes in cardiovascular/kidney function that could lead to increases in blood pressure, salt, and water retention, and decreases in blood levels of potassium and calcium
  • Behavioral and mood changes that could lead to potentially severe psychiatric adverse reactions
  • Osteoporosis, decrease in bone mineral density
  • Serious skin rashes
  • May slow growth and development
  • Cataracts or glaucoma

Vaccinations:  The administration of live or live attenuated vaccines is not recommended while taking EMFLAZA.  Killed or inactivated vaccines may be administered, but the responses cannot be predicted.  Patients should discuss their vaccine history with their healthcare provider before starting EMFLAZA, and while taking EMFLAZA, patients should check with their healthcare provider before receiving any new vaccines or booster shots.

Common side effects that could occur with 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.

Patients should tell their healthcare provider if they have had recent or ongoing infections, develop a fever, or experience any other side effects.

Please see for full Prescribing Information.

You may report side effects to ProPharma Group at 1-866-562-4620 or

You may report side effects to FDA at 1-800-FDA-1088 or

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Previous Article - Feb 13, 2017

An Open Letter to the Duchenne Community