Rocket Pharmaceuticals (RCKT) investor relations material

Introduction and purpose

• FDA granted accelerated approval to KRESLADI, the first gene therapy for severe LAD-I in pediatric patients, addressing an ultra-rare, life-threatening immunodeficiency with high early childhood mortality and limited treatment options.

• Approval makes the company eligible for a Rare Pediatric Disease Priority Review Voucher, supporting future pipeline development.

Details of approval or decision

• KRESLADI is approved for pediatric patients with severe LAD-I due to biallelic ITGB2 variants lacking an HLA-matched sibling donor.

• Approval was granted under the FDA's accelerated approval pathway based on biomarker restoration of neutrophil CD18 and CD11a expression; continued approval depends on confirmatory trials.

• The company now holds the PRV and is evaluating monetization options to extend its cash runway.

Impact on industry and stakeholders

• KRESLADI is the first and only gene therapy for severe LAD-I, marking a milestone for the severe LAD-I and broader primary immunodeficiency communities.

• Families and clinicians gain a new treatment option for a disease with limited alternatives.

• The company expects to treat a single-digit number of patients annually due to the ultra-rare nature of the disease.