Delandistrogene moxeparvovec

Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy. It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells. It is an adeno-associated virus vector-based gene therapy that is given by injection into a vein.

The most commonly reported side effects include vomiting, nausea, acute liver injury, pyrexia (fever), and thrombocytopenia (abnormally low platelet count in the blood).

Delandistrogene moxeparvovec was approved for medical use in the United States in June 2023. It was developed by Sarepta Therapeutics and is manufactured by Catalent.

Medical uses
Delandistrogene moxeparvovec is indicated for the treatment of ambulatory and non-ambulatory individuals four years of age and older with Duchenne muscular dystrophy with a confirmed mutation in the Duchenne muscular dystrophy gene.

Delandistrogene moxeparvovec is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin, a shortened protein (138 kDa, compared to the 427 kDa dystrophin protein of normal muscle cells) that contains selected domains of the dystrophin protein present in normal muscle cells. FDA states that the conditional approval is based on detection of successful gene expression; evidence of clinical improvement is still pending.

History
The accelerated US Food and Drug Administration (FDA) approval of delandistrogene moxeparvovec was based on data from a randomized clinical trial that established that delandistrogene moxeparvovec increased the expression of the Elevidys micro-dystrophin protein observed in delandistrogene moxeparvovec-treated individuals aged four to five years with Duchenne muscular dystrophy.

Economics
Initial pricing was announced at US$3.2 million for a single treatment which is expected to last lifetime.