Deflazacort Approved in the U.S. for Duchenne

UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (http://bit.ly/2kb1oCW).

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Today, the US Food and Drug Administration (FDA) approved EMFLAZA™ (deflazacort) for Duchenne. This is the first approval of a steroid for Duchenne and a therapy that applies to people with Duchenne 5 years of age and older, regardless of genetic mutation.

The history of deflazacort in the US

Until today, prednisone was used/recommended by physicians for their patients with Duchenne because clinical trial data suggested prednisone was able to slow progression and maintain the ability to walk for months to years. Clinical trials comparing another steroid, (deflazacort) with prednisone were initiated years ago, but the company who owned deflazacort was sold and the new company decided not to pursue the steroid in the US market. 

Deflazacort has been approved in several countries outside the US. Some families and physicians believe deflazacort has less impact on weight gain and less behavior related issues. For many years, those who could afford deflazacort paid out-of-pocket and had to figure out ways to access the medicine outside the US. While access improved over time, there were times when families found their supply of deflazacort held in customs and supply was not always assured. Sometimes, the impact on the family budget was too difficult to manage.

What does today's FDA approval of EMFLAZA™ (deflazacort) mean?

Today’s approval means that EMFLAZA is now available for all individuals diagnosed with Duchenne in the U.S.

Further, with FDA approval, the community is now assured that the product is CGMP (current good manufacturing practice). FDA regulates the quality of pharmaceuticals very carefully. CGMP refers to the Current Good Manufacturing Practice regulations enforced by the FDA. CGMPs provide for systems that assure proper design, monitoring, and control of manufacturing processes and facilities. Adherence to the CGMP regulations assures the identity, strength, quality, and purity of drug products by requiring that manufacturers of medications adequately control manufacturing operations.

With this approval, we now have an approved steroid, a pharmaceutical indicated for the treatment of Duchenne, and supply without worry.

Addressing questions

As news of Marathon Pharmaceutical’s interest and progress toward approval has been discussed over the last year, the question of out-of-pocket cost has been a top concern of many families. The label designation for individuals age 5 years and older poses additional questions about access for those under age 5.  

Representatives from Marathon will be joining us for a webinar on Friday, February 10th at 12 PM EST to provide information about EMFLAZA’s FDA-approved label, patient support services, and access to this medication – and to respond to questions from our community. See details on how to join us below.

UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (http://bit.ly/2kb1oCW).

Join our upcoming webinar with Marathon: Friday, February 10th, 2017

We are pleased to learn that the FDA has approved EMFLAZA (deflazacort). The FDA’s approval of EMFLAZA provides options for families when making decisions about care with their providers. During this webinar, the Marathon team will provide information about EMFLAZA’s FDA-approved label, patient support services, and access to this medication.

• Date: Friday, Feb. 10
• Time: 12 PM EST / 9 AM PST
• Speakers:

  • Pat Furlong, PPMD Founding President & CEO

  • Jeff Aronin, Marathon Pharmaceuticals CEO

  • Dr. Tim Cunniff, Marathon Pharmaceuticals, EVP of Research & Development

  • Eric Messner, Marathon Pharmaceuticals, VP of Sales & Marketing

  • Greg Wujek, Marathon Pharmaceuticals, VP of Market Access

• To Participate:

  1. Visit ReadyTalk.com and use access code 9449985. (Be sure to test your computer beforehand)
  2. Audio Dial-In Information:
     • U.S. & Canada: Dial 1.866.740.1260 and use the Access Code 9449985
     • Outside the U.S. and Canada: Lookup your number

This webinar is hosted by PPMD. In order to provide you with the most current information, this webinar will be recorded and made available on the PPMD website until Friday, February 24th, as directed by Marathon. After that time, please visit emflaza.com or call 1-844-EMFLAZA. As always, it is best to talk to your physician regarding your medical care.

Resources

• Emflaza.com
• EMFLAZA and Patient Support (PDF)
• Marathon's Press Release
• FDA's Press Release
• NDA Approval Letter (PDF)
• Label (PDF)
• View PPMD's Access Resources