Rocket Pharmaceuticals (RCKT) Shares Surge Following Landmark FDA Gene Therapy Approval

Summary

• The FDA has granted accelerated approval to KRESLADI, a gene therapy from Rocket Pharmaceuticals designed to treat children with severe leukocyte adhesion deficiency-I (LAD-I).
• Shares of RCKT surged by as much as 9% during premarket hours and maintained a gain of approximately 6% on Friday.
• KRESLADI represents the first gene therapy authorized by the FDA for severe LAD-I, a rare and fatal immune disorder in pediatric patients.
• Rocket was also issued a Rare Pediatric Disease Priority Review Voucher by the FDA, which the firm intends to sell for capital.
• This regulatory milestone follows a 2024 resubmission after the FDA initially turned down the application in June of that year to request more data.

(SeaPRwire) –   On Friday, the FDA greenlit Rocket Pharmaceuticals’ gene therapy KRESLADI, sparking a premarket rally in RCKT stock of up to 9% before it settled with a 6% increase at the opening bell.

Rocket Pharmaceuticals, Inc., RCKT

The therapy received accelerated approval based on surrogate endpoints, specifically the increased expression of CD18 and CD11a on the surface of neutrophils. Continued approval will be contingent upon long-term clinical evidence gathered from an ongoing trial and a post-marketing registry.

#NEWS: Rocket Pharmaceuticals today announced the U.S. FDA approval of the first #GeneTherapy for pediatric patients with severe Leukocyte Adhesion Deficiency-I (LAD-I).

More: https://t.co/YrJuWo1r3J pic.twitter.com/LkioRrrR5S

— Rocket Pharmaceuticals (@RocketPharma) March 27, 2026

KRESLADI, also referred to as marnetegragene autotemce, is a gene therapy utilizing a patient’s own hematopoietic stem cells. It is indicated for pediatric patients with severe LAD-I caused by mutations in the ITGB2 gene who do not have a matched sibling donor available for a stem cell transplant.

LAD-I is an exceptionally rare genetic immune deficiency that leads to chronic infections and high mortality rates in young children. In the United States, it is estimated to occur in one out of every 100,000 to 200,000 births, with severe forms accounting for roughly two-thirds of cases.

KRESLADI now stands as the inaugural FDA-approved gene therapy for this condition.

The path to this approval included hurdles; the FDA originally declined the biologics license application in June 2024, citing a need for additional data to finish the review process.

Rocket submitted the application again, and the FDA accepted it in October 2024, establishing a target action date of March 28, 2026—a deadline the agency successfully met.

Financial Upside from Rare Pediatric Disease Voucher

Alongside the approval, Rocket received a Rare Pediatric Disease Priority Review Voucher from the FDA. These vouchers are highly valuable assets that can be sold to other pharmaceutical firms for hundreds of millions of dollars, providing a significant financial benefit beyond the therapy’s market entry.

Rocket stated it is looking into options to monetize this voucher to increase its financial resources.

“The authorization of KRESLADI is a major achievement for the community affected by severe LAD-I,” remarked CEO Gaurav Shah.

Research Context

The development of KRESLADI was partially supported by funding from the California Institute for Regenerative Medicine. The treatment targets the ITGB2 gene, which is responsible for producing a protein that enables white blood cells to combat infections.

The biotechnology company, based in Cranbury, New Jersey, has been working toward this regulatory goal for several years.

The FDA finalized the approval of the resubmitted application exactly on the scheduled date of March 28, 2026.