We’re pleased to update that the US Food and Drug Adminisatration has expanded the approval of Elevidys for the treatment for ambulatory and non-ambulatory people with Duchenne muscular dystrophy who are four years of age and older.
FDA expand approval for Sarepta Therapeutic gene therapy Elevidys

Last year we reported that the US Food and Drug Administration’s (FDA) had granted accelerated approval for Sarepta Therapeutic’s gene therapy known as Elevidys, also known as delandistrogene moxeparvovec-rokl, for the treatment of ambulatory children (those able to walk unassisted) living with Duchenne muscular dystrophy (DMD) aged between four and five years old.
We’re pleased to update that the FDA has expanded the approval of Elevidys for the treatment for ambulatory and non-ambulatory people with DMD who are four years of age and older. You can read more about this on the FDA’s website.
As we mentioned before, the FDA’s decision will not directly affect people living with DMD in the UK because FDA decisions do not apply to the UK. We’re in contact with Roche – Sarepta’s partner for global distribution – who will provide more information for getting the treatment to people in the UK soon. The UK health authority responsible for issuing marketing authorisations (licenses) for medicines is the Medicines & Healthcare Products Regulatory Agency (MHRA) and not the FDA.
Roche is actively engaging with the MHRA to determine the best possible route to achieving a potential licence for Elevidys. In addition to gaining a licence, further assessments of clinical and cost-effectiveness by the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) must also be undertaken before a medicine is available to patients on the NHS. You can read more about this process on our Access to Treatments page.
DMD is a rare muscle wasting condition that mainly affects boys and men. People with DMD lack a protein called dystrophin, which is required to maintain the strength of muscles. Elevidys is a gene therapy for DMD developed by Sarepta Therapeutics. It’s based on the delivery of micro-dystrophin (a smaller, but functional, version of dystrophin protein) into muscle cells via an adeno-associated virus (AAV) vector.
We will keep you updated on developments that impact people in the UK, whenever we can.