US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

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Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.

FAQ – Eteplirsen for DMD

The experimental drug eteplirsen, under development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD), is under review by the U.S. Food and Drug Administration (FDA) for market approval. It marks an exciting and historic time for the MDA community and an important milestone for DMD treatments. Before a final decision is rendered, however, an FDA Advisory Committee will review the drug and provide a recommendation to the regulatory agency.

The advisory committee meeting for eteplirsen, originally scheduled for Jan. 22, 2016, was postponed on Jan. 20 due to Winter Storm Jonas and has now been rescheduled.

  • The meeting has been rescheduled to take place on April 25, 2016, from 8 a.m. to 6:30 p.m. ET.
  • The Ad Comm will take place at the College Park Marriott Hotel & Conference Center (a non-FDA campus venue) in order to accommodate what is anticipated to be large turnout.
  • The Prescription Drug User Fee Act (PDUFA) action date by which the FDA must make a decision on the drug is May 26, 2016.

What is an FDA Advisory Committee?

Part of the FDA mission is to evaluate new therapies and determine which are safe and effective for their intended use. Advisory committees provide the FDA with independent advice from outside experts on issues related to human and veterinary drugs, vaccines, medical devices, and food.

  • The Peripheral and Central Nervous System Drugs (PCNS) Advisory Committee reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of neurologic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs. One of 48 technical and scientific advisory committees, it is responsible for reviewing potential treatments for DMD.
  • In general, an advisory committee includes a chair and several members, plus a consumer, industry, and sometimes a patient representative.
  • Additional experts with special knowledge may be added to committee meetings as needed.
  • Although the committee provides advice to the agency, the FDA makes the final decision.
  • To learn more about FDA Advisory Committee Meetings, visit: Common Questions and Answers about FDA Advisory Committee Meetings.

Can I participate in the advisory committee for eteplirsen?

PLEASE NOTE: If you submitted written testimony last time or if you were scheduled for the open public hearing, you must resubmit for both.

You may participate in one of three ways:

  1. Written Testimony: Present data, information, or views, orally or in writing, by submitting them, before April 11 to: Moon Hee V. Choi, PharmD. Center for Drug Evaluation and Research. Food and Drug Administration, 10903 New Hampshire Ave., W031-2417, Silver Spring, MD 20993-0002. Phone: (301) 796-9001. Fax: (301) 847-8533. Email:
  1. Oral Testimony: Make an oral presentation at the committee meeting. Oral presentations will be scheduled between approximately 12:40 p.m. and 2:40 p.m. on April 25. If you are interested in making a formal oral presentation, please notify Moon Hee V. Choi (contact information above) on or before April 1, and submit a brief statement describing the comments you wish to make, the names and addresses of proposed participants, and an indication of the approximate time you will need to make your presentation.
  1. Participate as an audience member: Please visit the FDA website at Public Conduct During FDA Advisory Committee Meetings for procedures on public conduct during advisory committee meetings.

Will the advisory committee meeting be available for viewing online?

A free-of-charge, live webcast is planned for the meeting of the Peripheral and Central Nervous System Drugs Advisory Committee meeting.

  • Webcast information will be made available at least two days in advance of the meeting.
  • In addition, the webcast will be archived for viewing after the meeting concludes.

Background Information

What is eteplirsen?

Eteplirsen is an “exon skipping” drug in late-stage development for the treatment of DMD.

  • Administered by intravenous infusion, it targets a section of genetic code called “exon 51” in the dystrophin gene.
  • If approved by the FDA, it will be one of the first approved treatments designed to target the underlying cause of the disease.
  • Although not a cure, eteplirsen potentially could lessen the severe muscle weakness and atrophy that is the hallmark of DMD.

What is exon skipping?

Exon skipping is a strategy currently being developed for DMD in which sections of genetic code are “skipped” during the protein manufacturing process, allowing cells to create shortened but partially functional dystrophin protein — the muscle protein missing in DMD.

  • Laboratory development of exon skipping began in the 1990s, and MDA has funded extensive research into the strategy since that time.
  • MDA supported the early development of eteplirsen via funding to Steve Wilton, then at the University of Western Australia in Perth.
  • While it is being developed for DMD, exon skipping may have application to other genetic diseases down the line.

Will eteplirsen help my child?

It’s been estimated that 13 percent of boys with DMD may benefit from skipping exon 51.

  • Many families know that their child has a mutation in the dystrophin gene, but not all of them know exactly where the mutation is or what type it is.
  • Genetic testing is needed to pinpoint the exact nature and location of each individual’s dystrophin mutation and to determine whether or not a drug such as eteplirsen, that targets and “skips” exon 51, will be helpful.
  • For information, or to schedule genetic testing, please contact your child’s physician.

What about people with DMD that can’t be helped by skipping exon 51?

At this time, drug developers are working on therapies to target exons that are likely to help the greatest number of people.

  • Researchers currently are working on development of exon skipping drugs to target exons 44, 45, 50, 52, 53 and 55, with some working on strategies to target multiple exons.
  • DMD therapies based on various other strategies are under development as well.