Hemgenix

etranacogene dezaparvovec-drlb

Approval

ApplicationBLA 125772

Approval dateNov 22, 2022

Approval year2022

SponsorCSL Behring LLC

FDA-approved use

For treatment of adults with Hemophilia B (congenital Factor IX deficiency) who: Currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes

Approval package

↗ Drugs@FDA: full approval-package overview (every PDF the FDA released)
↗ FDA-approved label (PDF)

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 Hemgenix:

"What was the basis for Hemgenix's approval — what trials, what endpoints?"
"What adverse events did the FDA flag in the Hemgenix medical review?"
"What regulatory pathway did Hemgenix use — priority review, breakthrough, accelerated approval?"