Tag Archives: FDA

  1. An Open Letter to the Duchenne Community

    Posted on February 13, 201710:39 pm

      CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community Dear Members of the Duchenne Community: I want to thank you all for your support and encouragement over the last several years as Marathon worked to bring EMFLAZA™ (deflazacort) through the Read more »

  2. An Open Letter to the Duchenne Community

    Posted on 6:59 pm

      CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community  Dear Members of the Duchenne Community: I want to thank you all for your support and encouragement over the last several years as Marathon worked to bring EMFLAZA™ (deflazacort) through the Read more »

  3. FDA Approves EMFLAZA (deflazacort) … Our Experience With Deflazacort

    Posted on February 10, 20174:03 pm

      We are delighted that the U.S. Food and Drug Administration (FDA) has approved EMFLAZA (deflazacort) for the treatment of Duchenne. This is wonderful news for the Duchenne community to have access to this drug in the U.S. We are grateful for Marathon Pharmaceuticals for conducting the multiple studies necessary for FDA approval. When your Read more »

  4. Additional Information about EMFLAZA (deflazacort) for Duchenne Patients under the Age of Five

    Posted on 1:49 am

      Today, the FDA approved the use of EMFLAZA (deflazacort) for Duchenne patients over the age of five years old.  It is routine for the FDA to designate specific patient populations on the “label” that are approved to receive a drug.  The reason the FDA excluded younger patients for EMFLAZA is because there has not Read more »

  5. Deflazacort Transition Program Reminder

    Posted on January 17, 20176:42 pm

      Marathon submitted New Drug Applications (NDAs) to the Food and Drug Administration (FDA) last June for deflazacort. The NDAs have been accepted for filing and review. The FDA expects to complete its review of the applications in early February. If approved, deflazacort will become accessible in the U.S. and it would be the second Read more »

  6. Marathon Webinar: Update on Deflazacort Access and Regulatory Status

    Posted on November 4, 20164:48 pm

    Thank you to Marathon Pharmaceuticals for providing updated information about deflazacort access and regulatory status during a webinar this week.  The webinar addressed Marathon’s Expanded Access Program for deflazacort and what a potential FDA approval of deflazacort would mean in terms of access to the drug in the U.S.  The webinar has been archived.  https://cc.readytalk.com/play?id=hl12zw. Read more »

  7. Better Designed Clinical Trials Now

    Posted on October 31, 20168:25 pm

      As a parent of a son with Duchenne I have been frustrated that the data presented to the FDA does not match the patient experience.  I personally know so many patients who have been in recent clinical trials that are doing much better than expected and are seeing a slower progression of their disease Read more »

  8. First Drug Approved for Duchenne: A Beacon of Hope for the Duchenne Community

    Posted on September 20, 20165:08 pm

      My husband Paul and I have dreamed of this day since our son Hawken was diagnosed with Duchenne almost 15 years ago! We are overwhelmed with joy to announce that the FDA has officially granted accelerated approval for Eteplirsen for the treatment of Duchenne Muscular Dystrophy. This is the first approved drug-therapy and a Read more »

  9. BioMarin Update for the Duchenne Community: 01 June 2016

    Posted on June 2, 20162:59 pm

      We are sharing the following update from BioMarin. We would like to update the patient community in light of the recent announcement regarding the EMA marketing application. BioMarin is discontinuing clinical and regulatory development of drisapersen as well as the other three follow-on products: BMN 044, BMN 045 and BMN 053 Why have we Read more »

  10. Going Backwards to Go Forwards

    Posted on June 1, 201612:27 am

      I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe. Like you, I am heartbroken and sad to see this happen. From a business standpoint, BioMarin’s protective step makes sense. The company needs to regroup to find a better way forward. Read more »

  11. The Wait for An Approved Drug for Duchenne Continues

    Posted on May 25, 20161:57 pm

      The wait continues.  Sarepta Therapeutics announced this morning that the FDA will not complete the review of the eteplirsen new drug application by the PDUFA date.  Considering the thousands of lives that could be affected by a decision on eteplirsen, we understand and appreciate the care the FDA is taking in its review of Read more »

  12. Sarepta Advisory Committee Meeting

    Posted on April 22, 201611:45 pm

      As I prepare to travel to Monday’s Advisory Committee meeting I continue to be disappointed by the FDA Peripheral and Central Nervous System Drugs Advisory Committee’s initial review of Sarepta’s Duchenne muscular dystrophy drug eteplirsen. Despite this news we need to remember The FDA’s initial reaction is just that – an initial response – Read more »

  13. CureDuchenne Urges Regulators to Approve Eteplirsen, Stresses the Importance of Multiple Drugs in the Pipeline

    Posted on January 18, 20168:17 pm

      The Duchenne community continues its fight to get an approved treatment for Duchenne. The FDA released Briefing Document on eteplirsen, the drug for Duchenne muscular dystrophy submitted for approval by Sarepta Therapeutics Inc.  Although the review is harsh, we urge the Advisory Committee and the FDA to listen to the patient voice when making Read more »

  14. BioMarin DMD Update for Patient Groups

    Posted on January 15, 20167:02 pm

      We wanted to share the following information from BioMarin in regards to yesterday’s news on the FDA decision on Kyndrisa (drisapersen).     Dear Patient Group Representative, As you are likely aware, yesterday, we announced that the FDA issued a Complete Response Letter to BioMarin for our new drug application for Kyndrisa (drisapersen) for Read more »

  15. BioMarin Update to the Duchenne Community-November 25, 2015

    Posted on November 26, 201512:52 am

      BioMarin expresses its heartfelt appreciation to the Duchenne patient organizations, families, boys and young men living with Duchenne muscular dystrophy (Duchenne) who voiced their perspectives at the November 24th Food and Drug Administration (FDA) Advisory Committee meeting to consider our New Drug Application (NDA) for drisapersen for the treatment of Duchenne amenable to exon Read more »


    Posted on November 25, 201512:05 am

    CureDuchenne, the California-based nonprofit organization dedicated to finding cures for Duchenne muscular dystrophy, released the following statement from its co-founder and CEO, Debra Miller, following the meeting of the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA). The FDA will consider the committee’s discussions, and has set a Read more »

  17. FDA Advisory Committee Meeting

    Posted on November 20, 20154:50 pm

      We are looking forward to the U.S. Food & Drug Administration (FDA) Advisory Committee meeting on  Tuesday, November 24. This will be an important day for the Duchenne community. The committee members selected by the FDA are set to review clinical data for Drisapersen and hear testimony from patients, doctors and caregivers, the individuals Read more »

  18. BioMarin Quarterly Update to the Duchenne Community

    Posted on 12:47 am

      CureDuchenne is pleased to share the BioMarin Quarterly Update to the Duchenne Community.   It covers important information about next week’s FDA Advisory Committee meeting and other key updates from the company.     November 2015 BioMarin: Quarterly Update to the Duchenne Community Welcome to the second of the BioMarin quarterly updates. As you can Read more »

  19. You are Pioneers Once Again

    Posted on November 17, 20159:22 pm

      We are pleased to share the following letter from the CEO of PTC Therapeutics to the Duchenne community.  CureDuchenne funded PTC Therapeutics back in 2003 and we are proud to have helped advance the development of Translarna.  We look forward to PTC completing their rolling New Drug Application (NDA) with the Food & Drug Read more »

  20. Speak Up for the Duchenne Community!

    Posted on October 14, 201511:18 pm

      After waiting for a very long time, the Duchenne community now has clarity on the Advisory Committee meetings dates for BioMarin and Sarepta.  BioMarin’s date is November 24, 2015 and Sarepta’s date is January 22, 2016.  Please remember, these dates are tentative until they actually appear on the Federal Registry.  Both of the Ad Read more »

  21. Duchenne Community: Let Your Voice Be Heard!

    Posted on August 27, 20156:27 pm

      This week the FDA accepted Sarepta Therapeutics’ New Drug Application for eteplirsen with priority review. This follows on the June FDA’s acceptance of BioMarin’s NDA for drisapersen, also with priority review. The next step for both of these drugs is an FDA Advisory Committee meeting in November where both companies will demonstrate why these Read more »

  22. CureDuchenne’s Early Funding Projects Take a Big Step Forward Today

    Posted on June 29, 20153:58 pm

      FDA Accepts NDA for Drisapersen, the First-Ever Treatment for Duchenne Muscular Dystrophy to Reach this Milestone in the U.S., and Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen  It is a big day for the Duchenne community.  It is also a proud moment for CureDuchenne.  Today, BioMarin announced that the U.S. Food and Read more »

  23. Hope and Gratitude: Moving Closer to a Cure for Duchenne

    Posted on April 28, 20157:13 pm

    As I type this, my heart is filled with hope. A horizon that we could barely see 12 years ago has arrived. When our son Hawken was diagnosed with Duchenne Muscular Dystrophy 12 years ago, we felt lost and scared. By forming CureDuchenne we took on the challenge of finding a cure for this horrible Read more »

  24. FDA Webinar on Accelerated Approval Summary

    Posted on February 21, 20131:28 am

        CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases.   Dr. Robert Temple, director of the Office of Medical Policy of FDA’s Center for Drug Evaluation and Research presented detailed information on the different options available to speed the approval of Read more »

  25. CureDuchenne – Clinical Trials – FDA

    Posted on December 7, 201212:45 am

    Following the long awaited news of Sarepta’s phase IIB results for eteplirsen and continued Phase III progress of GSK/Prosensa’s drisapersen, as well as the recent developments with PTC Therapeutics and Summit PLC among others, CureDuchenne took the initiative to reach out for patients and advocacy groups alike to begin a dialogue with the FDA and Read more »