Rocket Pharmaceuticals, Inc. filed a Form 8-K with the SEC on September 15, 2026, disclosing that the company reached alignment with the U.S. Food and Drug Administration on key elements of the global pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease.

Key Highlights

  • FDA Alignment: The company announced alignment with the FDA on continued enrollment and dosing for the global pivotal Phase 2 trial of RP-A501.
  • Indication: RP-A501 is being studied for the treatment of Danon disease.
  • Recalibrated Dose: The FDA alignment includes a recalibrated dose as a key element of the trial design.
  • Pivotal Population: The trial is based on a 12-patient pivotal population, according to the filing.
  • Co-Primary Endpoints: Established 12-month co-primary endpoints are incorporated into the trial design.
  • Disclosure Date: The press release announcing the alignment was issued on September 15, 2026.

Rocket Pharmaceuticals Discloses FDA Alignment on RP-A501 Danon Disease Trial Design

According to the Form 8-K filed by Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) on September 15, 2026, the company issued a press release on that same date announcing alignment with the U.S. Food and Drug Administration on the structure of its global pivotal Phase 2 trial evaluating RP-A501 as a treatment for Danon disease. The filing was made under Item 7.01, Regulation FD Disclosure.

The filing states that the FDA alignment covers "continued enrollment and dosing and key elements of the global pivotal Phase 2 trial of RP-A501," specifically identifying three agreed-upon elements: a recalibrated dose, a 12-patient pivotal population, and established 12-month co-primary endpoints. No additional clinical or efficacy data were included in the 8-K text itself; the underlying details are contained in Exhibit 99.1, a press release incorporated by reference into the filing.

The company noted in the filing that the information disclosed under Item 7.01, including the exhibit, "shall not be deemed 'filed' for purposes of Section 18 of the Securities Exchange Act of 1934" and is not subject to the liabilities of that section. The filing was signed by Martin Wilson, General Counsel and Chief Corporate Officer of Rocket Pharmaceuticals, Inc., headquartered at 9 Cedarbrook Drive, Cranbury, NJ 08512.

What Was Disclosed

Rocket Pharmaceuticals filed an 8-K on September 15, 2026, reporting FDA alignment on continued enrollment, dosing, a recalibrated dose, a 12-patient pivotal population, and 12-month co-primary endpoints for its RP-A501 Danon disease Phase 2 trial.