CRANBURY - Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for KRESLADI (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.
This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression. Confirmation of clinical benefit will be based on the evaluation of longer-term follow-up data of treated patients in the ongoing clinical study and through a post-marketing registry.
With the approval of KRESLADI, the FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher (PRV), a program designed to encourage development of therapies for rare pediatric diseases. The Company intends to evaluate strategic options to monetize the PRV in a manner designed to enhance financial flexibility and maximize shareholder value.
'The approval of KRESLADI represents an important milestone for the severe LAD-I community,' said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. 'This approval reflects the dedication of patients, families, investigators, and regulators who have worked together to advance research of this ultra-rare disease. We look forward to making KRESLADI available to eligible patients in the United States.'
LAD-I is an ultra-rare genetic pediatric disease caused by mutations in the ITGB2 gene encoding for CD18, a key protein that is expressed along CD11 integrins to facilitate leukocyte adhesion to the blood vessel wall and migration to tissues to confine and clear infections and orchestrate wound repair. Patients with severe LAD-I typically show very diminished CD11a expression. Infants with severe LAD-I suffer from recurrent, life-threatening bacterial, and fungal infections that respond poorly to antimicrobials and require frequent hospitalizations. In the U.S., the incidence of LAD-I is estimated to range from approximately one in 100,000 to one in 200,000 live births, with roughly two-thirds of affected patients classified as having the severe form of the disease.
'As a clinician, I have seen firsthand the serious impact that severe LAD-I can have on young children and their families,' said Donald B. Kohn, M.D., Principal Investigator of the Phase 1/2 study and Distinguished Professor of Microbiology, Immunology & Molecular Genetics at the University of California, Los Angeles (UCLA). 'The approval of KRESLADI represents the culmination of many years of scientific research and clinical collaboration aimed at addressing the underlying cause of this devastating disease.'
'The approval of KRESLADI represents a significant development for individuals affected by severe LAD-I and the broader primary immunodeficiency community,' said Vanessa Tenembaum, Chief Executive Officer of the Jeffrey Modell Foundation, a global nonprofit organization dedicated to early diagnosis and treatments for primary immunodeficiency. 'For families impacted by this rare and serious disease this approval underscores the importance of continued efforts to improve outcomes for patients with primary immunodeficiencies.'
Research supporting the development of KRESLADI was made possible in part by funding from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480).
INDICATION AND USAGE
KRESLADI is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant.
This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression. Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated biotechnology company advancing gene therapies for rare and devastating cardiovascular diseases, with additional programs in hematology and immunology. Rocket's cardiovascular pipeline includes three clinical stage programs that each target one of the major inherited cardiomyopathy subtypes: hypertrophic, arrhythmogenic, and dilated cardiomyopathies. Together these conditions represent more than 100,000 patients in the U.S. and EU. The Company's platform is supported by proprietary AAV manufacturing capabilities, multi-year efficacy and safety data in cardiac gene therapy, and experience treating several cardiac patients across late-stage AAV programs.
Rocket Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as 'believe,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'look forward,' 'will give,' 'estimate,' 'seek,' 'will,' 'may,' 'suggest' or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's expectations regarding the expected commercial availability and launch timing of KRESLADI; anticipated patient enrollment and treatment timelines; market adoption and patient identification; Rocket's commercial strategy and launch readiness; Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates; the anticipated timing and impact of commercialization activities; Rocket's projected cash runway; potential monetization of the Rare Pediatric Disease Priority Review Voucher and the continued development and advancement of Rocket's pipeline programs. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, risks related to: the successful launch and commercialization of KRESLADI; the safety and efficacy profile of KRESLADI; the Company's ability to obtain and maintain regulatory approvals; the ability to confirm clinical benefit required for continued approval under the accelerated approval pathway; manufacturing and supply chain considerations, including reliance on third-party manufacturers; the availability and readiness of qualified treatment centers; payer coverage and reimbursement; patient identification and referral dynamics; the outcome of litigation; unexpected expenditures; Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting; Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled 'Risk Factors' in Rocket's Annual Report on Form 10-K for the year ended December 31, 2025, filed February 26, 2026 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Contact:
Meg Dodge
Email: mdodge@rocketpharma.com
Kevin Giordano
Email: media@rocketpharma.com
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