Health

Accelerated Approval of Elevidys (SRP-9001) for Children with DMD

Petition is directed to
Europäische Arzneimittel-Agentur (EMA)
30,337 supporters
87% achieved 35,000 for collection target
30,337 supporters
87% achieved 35,000 for collection target
  1. Launched June 2023
  2. Time remaining 6 days
  3. Submission
  4. Dialog with recipient
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11/29/2023, 08:43

Support for the gene therapy petition

Dear Sir or Madam

I hope this email reaches you in good health and good spirits. I am writing to you on behalf of our petition group, which is campaigning for rapid approval of Sarepta Therapeutics SRP-9001 (Elevidys) gene therapy in Germany.

As you may know, this gene therapy was approved in the USA almost six months ago. Since then, we have received more and more positive feedback from various publicly accessible forums supporting patients with Duchenne muscular dystrophy (DMD) about the effect of the gene therapy.

Some parents report that after 8 weeks of gene therapy administration, there are no more signs of the "Growers' sign", which is typical for boys with DMD and describes the difficulty in standing up and straightening the body. Others report that their children are able to climb stairs freely and unassisted after 10 weeks. In addition, some teachers have reported that the affected boys seem to have changed - they run, play on climbing towers and actively participate in the lives of other children. This information comes first-hand from parents whose children have already been treated with gene therapy.

Despite this positive feedback, the German media claimed that there had been no significant improvements. This is simply a false claim. Yes, the primary endpoint of study 3 was not met, but this may be due to the different courses of the disease. Furthermore, the assessment of the NSAA score is outdated. This assessment is based on exercises performed on single days. Boys with DMD have good and not so good days on which their strength varies. With the new assessment methods, data is collected over a longer period of time, similar to a fitness tracker (pedometer, etc.). In addition, the secondary endpoints of the study showed significant improvements that should not be ignored. Therefore, the observation period should be extended to assess the effect of this gene therapy over 5 to 10 years. There are data from study 1 that look very positive and show that the gene therapy has already had positive effects for almost 6 years and has no negative effects.

For these reasons, we continue to firmly believe in the positive benefits of this gene therapy and call for its rapid approval in Germany. So far, it is the only effective therapy for DMD. We therefore ask you to support our online petition and collect more signatures.

Thank you in advance for your support. Together we can make a positive difference and help those in need of this therapy.


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