Elevidys is the only approved gene therapy in the World for Duchenne muscular dystrophy and received accelerated approval in the US in June 2023, and is now approved in the United Arab Emirates, Qatar, Kuwait, Bahrain, Oman, Brazil and Israel for the treatment of ambulant children aged 4 through 5 years with Duchenne, who have a confirmed mutation in the DMD gene. [Read More: Roche announced that EMA has initiated review of the ELEVIDYS]
Sarepta Therapeutics announces expansion of US FDA approval in 2024 for Elevidys for Duchenne Muscular Dystrophy patients ages 4 and older.
Table of Contents
FDA Expanded Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy
After this announcement, U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene. [Read More: Sarepta Wins Full Approval and Label Expansion for DMD]
In Which Countries Is Elevidys Approved?
Elevidys gene therapy now approved in the US, United Arab Emirates, Qatar, Kuwait, Bahrain, Oman, Brazil and Israel.
Is Elevidys Approved In Europe?
Roche announces EMA has initiated review of the Elevidys Marketing Authorisation application for the treatment of Duchenne muscular dystrophy (DMD).
The EMA’s investigation is being followed in Europe and Turkey. Duchenne muscular dystrophy patients and their families are following the developments closely and are eagerly awaiting the day they will receive the drug.
Applications for approval are currently under review in Europe (EMA), Japan, Switzerland, Singapore, Hong Kong and Saudi Arabia. [Read More: Roche]
Learn More: Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy
Will EMA Not Approve Elevidys?
The European Medicines Agency (EMA) requested a temporary halt to Studies 104 (NCT06241950), Study 302 (ENVOL, NCT06128564) and Study 303 (ENVISION, NCT05881408) pending the completion of their investigation due to the death of a 16-year-old child who received Elevidys gene therapy. (Read More: Study 104, Study 302 and Study 303 were temporarily halted in Europe)
This announcement, which upset European families waiting for Elevidys to be approved, brought to mind the idea that the gene therapy will not be approved in the EMA region unless sufficient evidence is provided.
Discover More: Duchenne Muscular Dystrophy: Treatment & Cost
