A note from Debra

Prosensa is working towards long-term access to drisapersen

Posted on August 15, 2014 4:27 pm

CureDuchenne is pleased to share the latest update to patient groups from Prosensa, which outlines their progress to date, as well as future plans. CureDuchenne announced earlier in the week our $7 million collaboration with Prosensa to help speed access to treatments for Duchenne muscular dystrophy.

 

Dear Patient Group Representative,

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.

Read more.

 

 

 

Accelerating Access to Duchenne Drug Candidates

Posted on August 12, 20144:23 pm

Yesterday Prosensa and CureDuchenne announced an extension of our collaboration and a path to provide access to four experimental drugs for Duchenne patients. Ten years ago, CureDuchenne provided funding to Prosensa which allowed the company to move forward with their preclinical work and translate their exon skipping compounds into human clinical trials.  Since September 2013, Read more »

Dr. Flanigan’s Duplication 2 Duchenne Mutation – Nature Medicine

Posted on August 11, 20142:10 pm

We are very excited to see that Dr. Kevin Flanigan’s work on a rare Duchenne mutation, a duplication on exon 2, was published in Nature Medicine.  This prestigious magazine reported on two advances in his current work which was supported by CureDuchenne. The first was the successful creation of a new mouse model of Duchenne Read more »