Beqvez

fidanacogene elaparvovec-dzkt

Approval

ApplicationBLA 125786

Approval dateApr 25, 2024

Approval year2024

SponsorPfizer, Inc

FDA-approved use

For the treatment of adults with moderate to severe 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, and, Do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test

Approval package

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 Beqvez:

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