FDA Approves ELEVIDYS (delandistrogene moxeparvovec-rokl) Gene Therapy for Duchenne Muscular Dystrophy
Yesterday, the U.S. Food and Drug Administration (FDA) granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl). This is a micro-dystrophin adeno associated viral vector (AAV) gene therapy by Sarepta and Roche. This approval marks the first gene therapy approval for Duchenne muscular dystrophy (DMD), a rare genetic neurological disorder.
In a letter to members of the World Duchenne Organization, Roche explains the impact and next steps of this approval for families living in- and outside of the USA. We decided to share this important news with the global community in this news article.
Roche Community Letter to Members of the World Duchenne Organization
Approval process of ELEVIDYS by the FDA
The FDA’s decision to grant accelerated approval to ELEVIDYS is based on expression of micro-dystrophin in muscle of treated patients, which is expected to lead to future clinical benefit. In addition to micro-dystrophin data, extensive clinical data provided through the Biologics License Application (BLA) was submitted by Roche. This application included safety and efficacy data from three Phase 1 and Phase 2 studies: Study 101 (NCT03375164), Study 102 (NCT03769116), and Study 103 (ENDEAVOR, NCT04626674). Additionally, an integrated analysis was conducted across these studies to compare functional results with a propensity-score-weighted external control. This data package showcased the potential of delandistrogene moxeparvovec-rokl in treating DMD.
Inclusion criteria for delandistrogene moxeparvovec
ELEVIDYS has been specifically approved for the treatment of individuals between the ages of 4 and 5 who have been diagnosed with Duchenne muscular dystrophy and meet certain criteria. Eligible patients must be able to walk and should not have antibodies to AAVrh74, a viral vector used in the gene therapy process. If you live in the USA, and your child is ambulatory and 4-5 years of age, we encourage you to reach out to your neuromuscular specialist, to determine whether your child is eligible for ELEVIDYS. They will provide you with detailed eligibility criteria and guide you through the decision-making process.
Who is not eligible to receive delandistrogene moxeparvovec?
The therapy is only approved in the USA for ambulatory patients aged 4-5 years. So patients who do not live in the USA, or those under 4 or over 5 years old are not able to receive this treatment under the current approval. It’s important to note that individuals with specific DMD gene variants, including any deletion in exon 8 and/or exon 9, will not be eligible to receive ELEVIDYS due to safety concerns. Finally, eligible patients should not have antibodies to AAVrh74. A blood test is needed to assess whether this is the case. Your neuromuscular specialist will be able to provide you with more detailed information and address any concerns you may have regarding eligibility.
What will happen if you live in the USA?
For residents of the United States, Sarepta has established SareptAssist, a dedicated resource to assist with accessing approved therapies. If you have any questions or need guidance on how to access ELEVIDYS or other Sarepta therapies, please don’t hesitate to contact SareptAssist at 1-888-SAREPTA (1-888-727-3782) or via email at [email protected].
What will happen if you live outside the USA?
For individuals residing outside the United States, Roche is actively engaging with health authorities in various countries to facilitate the approval and availability of delandistrogene moxeparvovec-rokl. Countries that can accept applications based on Phase 1 and Phase 2 data include Bahrain, Brazil, Israel, Kuwait, Oman, Qatar, Saudi Arabia, Singapore, and the United Arab Emirates. Roche is committed to submitting applications to these health authorities as soon as possible.
In Europe, the European Medicines Agency (EMA) has indicated the importance of including clinical trial data from the Phase 3 EMBARK study for delandistrogene moxeparvovec in their assessment process. If the EMBARK data proves supportive, Roche intends to submit marketing authorization applications to the EMA and other relevant health authorities promptly.
For further details on the FDA approval of delandistrogene moxeparvovec and to gain insights from the community, we encourage you to read the Community Letter provided by Roche.
As a global patient organization, we remain committed to providing support, resources, and information to patients and their families, ensuring they stay informed about the latest developments in the field of Duchenne and Becker muscular dystrophy. We will continue to monitor the progress of delandistrogene moxeparvovec-rokl and keep you updated.
