Over a 52-week treatment period, the gene therapy demonstrated robust evidence for a clinically meaningful benefit, including across several prespecified functional secondary end points vs placebo.
The drug’s mode of action is different than other types of corticosteroids because it’s based on the differential effects on glucocorticoid and mineralocorticoid receptors.
Failing to meet the primary endpoint in its confirmatory phase III Embark trial, Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec), which received accelerated approval in June and was priced at $3.2 million, has one of three fates in its future, all of which are dependent on how the U.S. FDA perceives the data.
Less than two weeks after getting a thumbs-up from the EMA’s Committee for Medicinal Products for Human Use (CHMP), Santhera Pharmaceutical AG’s vamorolone secured U.S. FDA approval for use in patients, 2 and older, with Duchenne muscular dystrophy (DMD). A first-in-class drug, vamorolone, branded Agamree, is expected to offer a safer alternative to the steroid therapy, which the company has said will remain a foundational treatment of DMD, even with the introduction of gene therapies.
Sarah Boyce, president and CEO at Avidity Biosciences, discusses her leading role at the company, as well as antibody oligonucleotide conjugate drug, AOC 1044, currently in development for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping (DMD44).