Eteplirsen 50 mg/mL Suppliers & Bulk Manufacturers

Available Forms: Injection

Available Strengths: 50 mg/mL

Reference Brands: Exondys 51 (USA/EU)

Category: Neurology

Eteplirsen is an antisense oligonucleotide used to treat Duchenne Muscular Dystrophy (DMD) in patients with a confirmed mutation amenable to exon 51 skipping. It works by promoting the production of functional dystrophin protein, which is crucial for maintaining muscle strength and function. Eteplirsen is administered as an intravenous infusion, typically once a week.

Product Description:

Eteplirsen is a phosphorodiamidate morpholino antisense oligonucleotide for Duchenne muscular dystrophy caused by deletions amenable to exon 51 skipping — about thirteen percent of patients. By binding the dystrophin pre-mRNA it causes the splicing machinery to skip exon 51, restoring the reading frame and producing a shortened, partly functional dystrophin, turning a Duchenne-type mutation into something closer to the milder Becker phenotype.

It is given as a weekly intravenous infusion. Approval rested on an increase in dystrophin protein as a surrogate end point; the amount produced is very small, typically around one percent of normal, and whether it translates into a clinically meaningful benefit is still being tested in confirmatory trials. Tolerability has been good, with infusion reactions, headache and balance disturbance among the reported adverse effects. Eligibility depends on genetic confirmation of an amenable mutation.

Sourcing notes. Originator product on accelerated approval in the US; it has not been authorised in the European Union, where the marketing authorisation application was refused. Supply outside the US is therefore named-patient or special-access import. Annual cost is very high, and payers in several US states restrict coverage. No generic route exists. Genetic testing capability in the destination market is a prerequisite for any use.