Rocket Pharmaceuticals (RCKT) Stock Pops on Historic FDA Gene Therapy Win

TLDR

• The FDA granted accelerated approval for KRESLADI, Rocket Pharmaceuticals’ gene therapy for severe leukocyte adhesion deficiency-I (LAD-I) in children.
• RCKT stock jumped as much as 9% in premarket trading and settled around 6% higher on Friday.
• KRESLADI is the first FDA-approved gene therapy for severe LAD-I, a rare and life-threatening immune condition in children.
• The FDA also awarded Rocket a Rare Pediatric Disease Priority Review Voucher, which the company plans to monetize.
• The approval came after a resubmission following an FDA rejection in June 2024 that asked for more data.

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The FDA approved Rocket Pharmaceuticals’ gene therapy KRESLADI on Friday, sending RCKT up as much as 9% in premarket trading before settling around 6% higher by the open.

Rocket Pharmaceuticals, Inc., RCKT

The approval was accelerated, meaning it was granted based on early evidence — specifically increased neutrophil CD18 and CD11a surface expression. Full confirmation of clinical benefit will depend on longer-term data from an ongoing study and a post-marketing registry.

KRESLADI, also known as marnetegragene autotemce, is an autologous hematopoietic stem cell-based gene therapy. It is approved for pediatric patients with severe LAD-I caused by biallelic variants in the ITGB2 gene who do not have an available HLA-matched sibling donor for a stem cell transplant.

LAD-I is an ultra-rare genetic immunodeficiency. It causes recurrent infections and carries high early-childhood mortality without treatment. In the U.S., incidence is estimated at roughly one in 100,000 to one in 200,000 live births, with about two-thirds of those cases classified as severe.

KRESLADI is now the first FDA-approved gene therapy for this condition.

The approval didn’t come without a bumpy road. The FDA rejected the biologics license application in June 2024, asking Rocket to submit additional data before it could complete its review.

Rocket resubmitted the BLA, and the FDA accepted it in October 2024, setting March 28, 2026 as the target action date — a deadline the agency met.

Rare Pediatric Disease Voucher Adds Financial Upside

Along with the approval, the FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher. These vouchers can be sold to other pharmaceutical companies for hundreds of millions of dollars and are seen as a meaningful financial bonus on top of the approval itself.

Rocket said it plans to evaluate options to monetize the voucher to boost financial flexibility.

Background on the Research

Research supporting KRESLADI’s development was funded in part by the California Institute for Regenerative Medicine. The drug targets the ITGB2 gene, which encodes a protein essential for white blood cells to fight infection.

The Cranbury, New Jersey-based biotech has been working toward this approval for several years.

The FDA’s action date for the resubmitted BLA was March 28, 2026, and the agency approved the therapy right on schedule.

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