---
schema_version: "secwatch.filing_event.v1"
accession: "0001140361-23-043687"
form_type: "8-K"
ticker: "RCKT"
cik: "0001281895"
company_name: "ROCKET PHARMACEUTICALS, INC."
filed_at: "2023-09-12T23:59:59+00:00"
generated_at: "2026-06-10T19:59:26.196706+00:00"
event_type: "regulatory"
sentiment: "positive"
materiality_score: 0.75
calibrated_materiality_score: 0.75
confidence: "high"
source: SEC EDGAR
---

# Rocket Pharma reaches FDA alignment on Phase 2 pivotal trial for RP-A501 in Danon Disease

## Summary
- FDA agreed to single-arm, open-label trial with 12 patients, natural history comparator, and co-primary endpoint of LAMP2 expression and LV mass at 12 months for accelerated approval.
- In-house cGMP manufacturing completed with sufficient drug product to supply the pivotal study; potency assays qualified per FDA guidance.
- ICD-10 code secured from CMS for LAMP2 deficiency; CTA/IMPD filing for EU initiation expected by end of Q3 2023.
- RP-A501 is a one-time infusion targeting a uniformly fatal cardiomyopathy with no approved therapies; affects 15,000–30,000 in US and Europe.

## SEC filing metadata
- accession: 0001140361-23-043687
- form_type: 8-K
- ticker: RCKT
- cik: 0001281895
- company_name: ROCKET PHARMACEUTICALS, INC.
- filed_at: 2023-09-12T23:59:59+00:00
- event_type: regulatory
- sentiment: positive
- materiality_score: 0.75
- calibrated_materiality_score: 0.75
- confidence: high
- sec_items: 7.01, 8.01, 9.01
- EDGAR index: https://www.sec.gov/Archives/edgar/data/1281895/000114036123043687/0001140361-23-043687-index.htm
- EDGAR primary document: https://www.sec.gov/Archives/edgar/data/1281895/000114036123043687/ny20010507x2_8k.htm

## Machine-readable alternates
- HTML: https://secwatch.observer/filing/0001140361-23-043687
- JSON: https://secwatch.observer/filing/0001140361-23-043687.json
- Plain text: https://secwatch.observer/filing/0001140361-23-043687.txt

This AI-assisted summary is a reading aid. Review the linked SEC EDGAR filing before relying on any specific claim.