PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne in the U.S.

Today, PTC Therapeutics announced that they have completed their acquisition of Emflaza™ (deflazacort) ahead of schedule, following early conclusion of the anti-trust review.

PTC will be hosting a call with the Duchenne community the week of May 8 to provide details on the plan for the launch of Emflaza. We look forward to learning more from PTC in the coming weeks and will keep you posted on call-in details. 

Read PTC's Letter to the Duchenne Community: 

Dear Duchenne Community,

Today we announced that we have completed our acquisition of EmflazaTM (deflazacort), the first FDA-approved, anti-inflammatory therapy for Duchenne Muscular Dystrophy (DMD) patients age 5 and older regardless of the genetic mutation. We are pleased the acquisition completed ahead of schedule, following early conclusion of the antitrust review period. During the week of May 8th, we will be hosting a call to provide you and everyone in the Duchenne community an update on the plans for launching Emflaza. We will share the conference call details with you once they are finalized.

We have been having conversations with you and other Duchenne stakeholders. These discussions have been invaluable as we develop our plans, and we are grateful for your insights. Our goal for the update call will be to give you an outline of our plans and hopefully answer many of your questions regarding access and impact to your healthcare benefits overall. As we develop our plans, our goal is to enable access to Emflaza for eligible patients, regardless of insurance status. This commitment extends to the work we’re doing internally to build programs that provide comprehensive patient assistance and support.

All of us at PTC are proud of our long-standing relationships and partnerships with the Duchenne community. We thank you again for your support during this transition. We are excited to work with you to bring this therapy to U.S. Duchenne patients. We hope you can join us on the Emflaza update conference call the week of May 8th where we will share details of our plans, including pricing and access programs. We will be in touch to share the conference call details.


Stuart Peltz 

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Read the Press Release from PTC:

PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne Muscular Dystrophy in the U.S.

SOUTH PLAINFIELD, N.J., April 20, 2017 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced it has completed its acquisition of all rights to Emflaza™ (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) in the U.S. Execution of the asset purchase agreement setting forth the terms of the acquisition was announced on March 16, 2017.

"We are pleased the acquisition was completed ahead of schedule, following early conclusion of the antitrust review period," stated Stuart Peltz, Ph.D., chief executive officer, PTC Therapeutics, Inc. "We've been engaging with key stakeholders in the DMD community to understand their needs and are working to make Emflaza commercially available as soon as possible. PTC is committed to bringing this important therapy to patients with DMD. Our goal is to enable access for eligible patients irrespective of insurance status, and we look forward to discussing commercial launch details on our upcoming quarterly earnings call."

Financial terms of the acquisition include a total upfront consideration of $140 million paid to Marathon Pharmaceuticals. Marathon is also entitled to receive payments from PTC based on annual net sales of Emflaza beginning in 2018, which PTC expects will range as a percentage of net sales between the low to mid-20s on a blended average basis. In addition, Marathon has the opportunity to receive a single $50 million sales-based milestone.

About Duchenne Muscular Dystrophy 
Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD can lose the ability to walk as early as age ten, followed by loss of the use of their arms. DMD patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. More information regarding DMD is available through the Muscular Dystrophy Association and the Parent Project Muscular Dystrophy. Additionally, information and resources are available at

About PTC Therapeutics 
PTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of novel medicines using our expertise in RNA biology. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. Since its founding nearly 20 years ago, PTC's mission has focused on developing treatments to fundamentally change the lives of patients living with rare genetic disorders. The company was founded in 1998 and is headquartered in South Plainfield, New Jersey. For more information on the company, please visit our website

About Emflaza
EMFLAZA™ is indicated for the treatment of Duchenne muscular dystrophy in patients 5 years of age and older.

Contraindication: Do not use if you are allergic to deflazacort or any of the inactive ingredients in EMFLAZA.

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.

  • Hyperglycemia: Corticosteroids can increase blood glucose, worsen pre-existing diabetes, predispose those on long-term treatment to diabetes mellitus, and may reduce the effect of anti-diabetic drugs. Monitor blood glucose at regular intervals. For patients with hyperglycemia, anti-diabetic treatment should be initiated or adjusted accordingly.
  • Increased Risk of Infection: Tell your healthcare provider if you have had recent or ongoing infections or if you have recently received a vaccine or are scheduled for a vaccination. Seek medical advice at once should you develop fever or other signs of infection, as some infections can potentially be severe and fatal. Avoid exposure to chickenpox or measles, but if you are exposed, medical advice should be sought without delay.
  • Alterations in Cardiovascular/Kidney Function: EMFLAZA can cause an increase in blood pressure, salt and water retention, or a decrease in your potassium and calcium levels. If this occurs, dietary salt restriction and potassium supplementation may be needed.
  • Behavioral and Mood Disturbances: There is a potential for severe behavioral and mood changes with EMFLAZA and you should seek medical attention if psychiatric symptoms develop.
  • Effects on Bones: There is a risk of osteoporosis or decrease in bone mineral density with prolonged use of EMFLAZA, which can potentially lead to vertebral and long bone fractures.
  • Effects on Growth and Development: Long-term use of corticosteroids, including EMFLAZA may slow growth and development in children.
  • Ophthalmic Effects: EMFLAZA may cause cataracts or glaucoma and you should be monitored if corticosteroid therapy is continued for more than 6 weeks.
  • Vaccination: The administration of live or live attenuated vaccines is not recommended. Killed or inactivated vaccines may be administered, but the responses cannot be predicted.
  • Serious Skin Rashes: Seek medical attention at the first sign of a rash.
  • Drug Interactions: 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.

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.

Please see the accompanying 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

Read PTC's full press release.

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