Rocket Pharma (RCKT) gets FDA date; PRV eligible if approved

Rhea-AI Filing Summary

Rocket Pharmaceuticals (RCKT) announced FDA acceptance of its resubmitted BLA for KRESLADI (marne‑cel), an investigational lentiviral gene therapy for severe Leukocyte Adhesion Deficiency‑I (LAD‑I). The agency set a PDUFA date of March 28, 2026, establishing the formal timeline for a U.S. approval decision.

KRESLADI targets a rare, life‑threatening immune disorder that often proves fatal in childhood without allogeneic stem cell transplant. If approved, the company is eligible for a Rare Pediatric Disease Priority Review Voucher, which can carry strategic value for future regulatory filings. The company attached a press release (Exhibit 99.1) and cautioned that approval is not assured, consistent with forward‑looking statement disclosures.

Insights

Biotech regulatory analyst

FDA accepted the BLA resubmission and set a PDUFA date.

Rocket Pharmaceuticals reported the FDA accepted its resubmitted BLA for KRESLADI, with a PDUFA date on March 28, 2026. Acceptance confirms the filing is administratively complete and begins the formal review clock, a key milestone for a rare disease gene therapy.

The therapy addresses severe LAD‑I, where unmet need is high. The company notes eligibility for a Rare Pediatric Disease PRV if approved, which can be strategically valuable. However, acceptance does not imply approval; review outcomes can vary based on efficacy, safety, and manufacturing considerations.

Next catalyst is the March 28, 2026 decision date. Any future disclosures may clarify review classification, information requests, or advisory committee plans, which would frame regulatory risk and timing.

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, DC 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): October 14, 2025

Rocket Pharmaceuticals, Inc.

(Exact name of registrant as specified in its charter)

Delaware	001-36829	04-3475813
(State or other jurisdiction of incorporation)	(Commission File Number)	(IRS Employer Identification No.)

9 Cedarbrook Drive, Cranbury, NJ		08512
(Address of principal executive offices)		(Zip Code)

Registrant’s telephone number, including area code: (646) 440-9100

Not applicable

(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2):

☐
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common stock, $0.01 par value	RCKT	The Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 8.01. Other Events.

On October 14, 2025, Rocket Pharmaceuticals, Inc. (the “Company”) announced that the U.S. Food and Drug Administration (the “FDA”) has accepted resubmission of the Biologics License Application (BLA) for KRESLADI™ (marnetegragene autotemcel; marne-cel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant. The PDUFA date set by the FDA is March 28, 2026. The Company is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), should KRESLADI™ be approved.

A copy of the press release issued in connection with the announcement is attached as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated into this report by reference.

Forward-Looking Statements

Except for the factual statements made herein, information contained in this Current Report on Form 8-K consists of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects or future events, as well as words such as “believes,” “intends,” “expects,” “plans” and similar expressions, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Such forward-looking statements are not guarantees of performance and actual actions or events could differ materially from those contained in such statements. For example, there can be no assurance that KRESLADI™ will be approved by the FDA or that the Company will receive a PRV. Reference is also made to other factors detailed from time to time in the Company’s periodic reports filed with the Securities and Exchange Commission, including the Company’s most recent Annual Report on Form 10-K and any subsequent Quarterly Reports on Form 10-Q. The forward-looking statements contained in this Current Report on Form 8-K speak only as of the date of this Current Report on Form 8-K and the Company assumes no obligation to publicly update any forward-looking statements to reflect changes in information, events or circumstances after the date of this Current Report on Form 8-K, unless required by law.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits.

99.1	Press Release of Rocket Pharmaceuticals, Inc. dated October 14, 2025.
104	Cover Page Interactive Data File (embedded within the Inline XBRL document).

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

	Rocket Pharmaceuticals, Inc.
Date: October 14, 2025	By:	/s/ Martin Wilson
		Martin Wilson
		General Counsel and Chief Corporate Officer, SVP

FAQ

What did Rocket Pharmaceuticals (RCKT) announce?

The FDA accepted the resubmitted BLA for KRESLADI (marne‑cel), an investigational gene therapy for severe LAD‑I.

What is the PDUFA date for RCKT’s KRESLADI?

The FDA set the PDUFA date for March 28, 2026.

What condition does KRESLADI target?

KRESLADI targets severe Leukocyte Adhesion Deficiency‑I (LAD‑I), a rare genetic immune disorder.

Is Rocket Pharma eligible for a Priority Review Voucher (PRV)?

The company is eligible for a Rare Pediatric Disease PRV if KRESLADI is approved.

What document includes more details on the announcement?

A press release is attached as Exhibit 99.1.

Does BLA acceptance guarantee FDA approval for RCKT?

No. The company cautioned that there is no assurance of approval.