PDUFA Risk Dossier: Rocket Pharmaceuticals (RCKT) CMC Precedent Briefing for the March 2026 FDA Decision

This dossier is a short regulatory risk briefing on Rocket Pharmaceuticals’ gene therapy Kresladi, ahead of its March 28, 2026 PDUFA decision.

Kresladi’s clinical efficacy is effectively de-risked (100% overall survival in Phase 2), yet the asset trades at a discount following a CMC-driven Complete Response Letter (CRL) issued in June 2024.

The core question this briefing addresses:

When the FDA issues a CMC-driven CRL for a gene therapy with strong clinical data, does precedent suggest a procedural delay or a structural reset?

This dossier answers that question by analyzing how similar FDA disputes have historically resolved.

What’s inside

• Decision context: what the FDA objected to — and what it did not
• CMC precedent analysis (bluebird bio, BioMarin, Regeneron)
• Signal guide for interpreting FDA decision language
• Identification of unobservable risks (CDMO inspection exposure)

Length: ~6 pages (PDF)

What this is not

• Not an approval prediction
• Not an investment recommendation
• Not financial or legal advice

Who this is for

Biotech investors, analysts, and operators evaluating FDA execution risk in gene therapy.