Exon-Skipping Trial Updates from Sarepta & Prosensa


In the wake of promising exon-skipping trial announcements earlier this summer, PPMD has been fielding many questions from the community about upcoming trials. We reached out to both Sarepta and Prosensa for updates to share.

Sarepta Update

Sarepta provided the Duchenne patient community with a written update and webinar on their clinical program on Thursday, August 7. Regular updates on their clinical programs will also be shared through the online educational resource, Let’s Skip Ahead. Any questions you have may be addressed to Kara Boniface at kboniface@sarepta.com.

Prosensa Update

Prosensa provided the update below, with the promise of a more detailed update to the community coming soon (EDIT 8/15/2104: see additional update below):

Dear PPMD,

For the re-dosing program, we are on track for re-dosing the first group of boys this quarter. As communicated earlier, in North America eligible boys are those who completed DEMAND V (DMD114876), those that are currently in the DMD115501 protocol and US/Canadian boys who participated in the DEMAND IV study (DMD114349). Subjects will initially be dosed in the clinic for the first 4 weeks, but home dosing is intended, subject to site requirements. No biopsies will be taken under this protocol. More detailed information about the re-dosing protocol for the first group of boys in Europe is expected soon.

We have added a dedicated page to the Prosensa website at www.prosensa.eu/patients-and-family/drisapersen-re-dosing, to enable easier tracking of the latest regarding drisapersen re-dosing information that is publicly available. This page also contains the latest patient group communications, which provide updates on Prosensa’s development programs for Duchenne and the latest webinar for the patient community. Of course for families and boys that participated in the drisapersen studies, their primary point of contact is the physician at the trial site, but we felt it would be helpful to have all information that is publicly available at one place.

As you are aware, Prosensa received guidance from the FDA on June 2nd, in which a clear regulatory pathway was outlined for drisapersen. Based on this guidance, we communicated that we intend to submit a file to the FDA later this year and will commit to the initiation of two confirmatory post-approval studies, as recommended by the FDA (to be commenced prior to an approval). One of these studies will be an open-label study with drisapersen (where the results will be compared with natural history control group) and the other with one of our other exon-skipping investigational compounds for DMD (which will be a placebo controlled study). Prosensa is working hard to finalize the study design of these confirmatory studies. Therefore, there is no information available yet about participating sites, inclusion and exclusion criteria, etcetera. I will make sure to clarify timelines for all of our investigational compounds in our next update.


If families have questions for Prosensa please have them reach out to patientinfo@prosensa.nl.

Additional Prosensa Update on August 15th

As previously communicated, Prosensa received guidance from the United States Food and Drug Administration (FDA) on June 2, in which a regulatory pathway for accelerated approval was outlined for drisapersen. Based on this guidance, we communicated that we intend to submit a file to the FDA later this year and will commit to the initiation of two confirmatory post-approval studies, as recommended by the FDA (to be commenced prior to an approval). We also have ongoing interactions with the European Medicines Agency (EMA), and we anticipate filing for a conditional approval in the EU shortly after the submission of a New Drug Application to the FDA. We are encouraged by the flexibility recently shown by both FDA and EMA, regarding products for DMD.

Prosensa is currently focusing on the preparation of the New Drug Application for submission to the FDA later this year. This is always a monumental undertaking, as New Drug Applications are typically made up of about 100,000 pages which display the results of all of the available clinical and pre-clinical trials as well as the analysis and interpretation of this data. Information about the manufacturing and quality control of the drug is also included.

One of the confirmatory post-approval studies will be an open-label study with drisapersen where the results will be compared with a natural history control group. A Prosensa natural history study is already ongoing and fully recruited with 269 participants. The other confirmatory post-approval study for drisapersen will be a placebo-controlled study with one of our other exon-skipping investigational compounds for DMD. For this study Prosensa will choose one product out of its clinical products PRO044, PRO045 and PRO053.Prosensa is working hard to finalize the study design of the confirmatory studies. We anticipate commencing both confirmatory studies in the first half of 2015 and will have more information on the study design and protocols in the coming months.



PRO052 and PRO055 are in advanced preclinical development. PROSPECT is in early pre-clinical development and applies multiple exon skipping focused on the exon 10-30 region.

For the drisapersen re-dosing program, we are on track for re-dosing the first boys prior to the end of September. The re-dosing protocols for this group have been finalized both for North America and Europe, sent to the relevant health authorities and we have begun the process of submitting to the relevant Institutional Review Boards (IRBs) at the hospitals of the participating sites for local ethics approval. For individual centers the starting date will also be dependent on the time it takes to gain the necessary IRB approval.

As communicated earlier, in North America eligible boys are those who completed DEMAND V (DMD114876), those that are currently in the DMD115501 protocol and US/Canadian boys who participated in the DEMAND IV study (DMD114349). In the first week of August, Prosensa organized an investigator meeting for the participating sites, which aimed to discuss the final protocol and logistics for conducting the study. We anticipate to start entering boys into the re-dosing program early September and to re-dose the boys from the first sites to get IRB approval around mid- September. The burden of participating in the open label re-dosing program is significantly lower for boys and their families compared to the original studies. Subjects will initially be dosed in the clinic for the first 4 weeks, but home dosing is intended, subject to site requirements. Since the primary aim of the re-dosing program is to provide access to drisapersen, no biopsies will be taken under this protocol.

In Europe, eligible boys are those that participated in the long-term CLIN-02 study (DMD114673). Pending final regulatory approvals, re-dosing for this first group is also scheduled to begin before the end of September and will be done under the existing treatment protocol. An amended protocol will be used to reduce the burden for participating boys and their families. As communicated before, re- dosing of boys that participated in drisapersen studies will take a staged approach. Prosensa intends to provide access for all patients that previously participated in the drisapersen studies, provided safety criteria are met. We are looking into the possibilities for re-dosing of the remaining studies on a country by country basis and are currently working on expected timelines for the next group of countries.

A dedicated page at the Prosensa website www.prosensa.eu/patients-and-family/drisapersen-re-dosing enables easier tracking of the latest information regarding the drisapersen re-dosing program that is publicly available. Families and patients that have previously participated in drisapersen clinical trials are encouraged to remain in contact with the investigator at their site, who is their primary point of contact. 

More Pipeline Updates

PPMD will continue to request the latest updates from all companies as drugs move through the pipeline. View our Research FAQ Sheets.

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