Marathon Pharmaceuticals to Begin NDA Process for Deflazacort as a Potential Treatment for Duchenne

Last week, Marathon Pharmaceuticals announced that the company will begin the New Drug Application (NDA) process for deflazacort as a potential treatment for patients with Duchenne following a positive pre-NDA meeting with FDA.  The deflazacort NDA is expected to be submitted in the first quarter of 2016 and will be subject to review by the FDA. If approved, deflazacort is expected to be made commercially available in the U.S. in the first quarter of 2017. The FDA previously granted Fast Track status and Orphan Drug designation for deflazacort for the treatment of patients with Duchenne.

 

Marathon has requested authorization from the FDA to provide deflazacort as an investigational* drug to qualified patients with Duchenne through an expanded access program (EAP) – at no cost to patients during the length of the EAP. FDA indicated that it may formally authorize this program pending a full review of the EAP protocol.  If the program is authorized by FDA, doctors will be able to enroll qualified patients to receive this investigational* medication through the EAP beginning in September 2015.

 

Click here to read the full press release. To learn more about Marathon’s Duchenne program, watch their presentation at PPMD’s 2015 Connect Conference. PPMD will be hosting a webinar with Marathon sometime next month.

 

Patients , families, and physicians can receive notifications of Marathon’s clinical trials in Duchenne by visiting AccessDMD.com.

*investigational medications have not yet been approved by the FDA, and they have not been proven to be safe and effective.

Read the Press Release:

Marathon Pharmaceuticals to Begin New Drug Application Process for Deflazacort as a Potential Treatment for Patients with Duchenne Muscular Dystrophy

Northbrook, Ill. – August 05, 2015 – Marathon Pharmaceuticals, a biopharmaceutical company that develops new treatments for rare diseases, today announced that the company will begin the New Drug Application (NDA) process for deflazacort as a potential treatment for patients with Duchenne muscular dystrophy (DMD) following a positive pre-NDA meeting with the U.S. Food and Drug Administration (FDA).

DMD is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking, breathing and ultimately death.1 Diagnosis typically occurs between the ages of 2 and 5 with progressive weakness leading to a loss of ambulation in the pre-teen to teenage years. Though DMD affects approximately 15,000 boys in the United States, there are currently no approved therapies for the disease in the U.S.2

The deflazacort NDA is expected to be submitted in the first quarter of 2016 and will be subject to review by the FDA. If approved, deflazacort is expected to be made commercially available in the U.S. in the first quarter of 2017. The FDA previously granted Fast Track status and Orphan Drug designation for deflazacort for the treatment of patients with DMD.

Marathon has requested authorization from the FDA to provide deflazacort as an investigational drug* to qualified patients with DMD at no cost, through an expanded access program (EAP). FDA indicated that it may formally authorize this program pending a full review of the EAP protocol. If the program is authorized by FDA, doctors will be able to enroll qualified patients to receive this investigational* medication beginning in September 2015.

Patients, families and physicians can receive notifications of Marathon’s clinical trials in DMD by visiting http://www.AccessDMD.com.

The development of deflazacort is part of a broader effort by Marathon to provide new treatment options for patients with DMD and other rare diseases. Additionally, Marathon is developing a national precision medicine program in partnership with leading patient advocacy, research and commercial organizations to accelerate research on treatments for DMD. This effort will launch in the first quarter of 2016.

*investigational medications have not yet been approved by the FDA, and they have not been proven to be safe and effective.

Watch Marathon's Presentation From This Year's Connect Conference:

Receive Notifications of Marathon's Clinical Trials in Duchenne: 

Click here to sign up.

 

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