Elevidys (delandistrogene moxeparvovec-rokl) FDA approval

FDA-approved use

ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who are ambulatory and have a confirmed mutation in the DMD gene.Limitations of Use:ELEVIDYS is not recommended in patients with:Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limitof normal or total bilirubin > the upper limit of normal not due to Gilbert’s syndrome) or activehepatic viral infection due to the high risk of acute serious liver injury and acute liver failure.Recent…

Query with Claude

The fda-analyst skill answers natural-language questions about this approval by fetching the relevant FDA review PDFs at query time.

git clone https://github.com/cbetz/trove cp -r trove/skills/fda-analyst ~/.claude/skills/

Example prompts for Elevidys:

"What was the basis for Elevidys's approval — what trials, what endpoints?"

"What adverse events did the FDA flag in the Elevidys medical review?"

"What regulatory pathway did Elevidys use — priority review, breakthrough, accelerated approval?"

Elevidys

Approval

FDA-approved use

Approval package

Query with Claude