Rocket Pharma Jumps 10% After FDA Approves First-Ever LAD-I Therapy

FDA Greenlights Kresladi, Sending RCKT Stock Up 10%

Rocket Pharmaceuticals (RCKT) shares increased 10% in pre-market trading Friday after the U.S. Food and Drug Administration approved its gene therapy, Kresladi, on March 27, 2026. The therapy is the first-ever approved treatment for severe leukocyte adhesion deficiency type I (LAD-I), a rare and often fatal immune disorder that affects children. Kresladi is specifically approved for pediatric patients who lack a matched sibling donor for a stem cell transplant, the previous standard of care.

The therapy, known chemically as marnetegragene autotemcel, is administered as a single intravenous dose. It is created by genetically modifying a patient's own hematopoietic stem cells to introduce a functional copy of the ITGB2 gene, which is defective in LAD-I patients. This landmark approval provides a critical treatment option for a disease that has a mortality rate of approximately 75% within the first two years of life.

Approval Follows June 2024 Manufacturing Rejection

The regulatory approval marks a significant turnaround for Rocket Pharmaceuticals. The FDA had initially rejected Kresladi in June 2024, citing a need for more information regarding the company's manufacturing processes. Rocket refiled its application in October 2024 after addressing the agency's concerns. This successful second attempt de-risks the company's manufacturing platform and validates its strategic focus.

This victory follows a deliberate strategic shift by the company, which previously withdrew its marketing application for RP-L102, a gene therapy for Fanconi anaemia. The company stated the withdrawal was for commercial reasons, allowing it to concentrate its resources on bringing Kresladi to market.

Rocket Gains $200M Asset with Priority Review Voucher

While Kresladi is expected to carry a multi-million dollar price tag, its direct revenue potential is limited by the disease's rarity, which affects an estimated one in a million people. However, the FDA approval delivers a more immediate financial benefit: a transferable Priority Review Voucher (PRV). These vouchers can be sold to other pharmaceutical companies for sums up to $200 million, providing a substantial source of non-dilutive capital. The strong clinical data, showing 100% patient survival at the two-year mark in trials, provides a powerful case for both pricing and reimbursement from insurers.