Rocket Pharmaceuticals (NASDAQ:RCKT) announced that the US Food and Drug Administration (FDA) has granted accelerated approval for KRESLADI (marnetegragene autotemcel), the company’s gene therapy for children with severe leukocyte adhesion deficiency-I (LAD-I) caused by biallelic variants in ITGB2.
The therapy is intended for patients without an available human leukocyte antigen-matched sibling donor for allogeneic stem cell transplant.
The approval, based on increased neutrophil CD18 and CD11a surface expression, is conditional and requires confirmation of long-term clinical benefit through ongoing studies and a post-marketing registry.
Alongside the approval, Rocket received a Rare Pediatric Disease Priority Review Voucher (PRV), which the company may monetize to enhance financial flexibility.
Severe LAD-I is an ultra-rare genetic immunodeficiency that leads to recurrent, life-threatening infections in early childhood. Incidence in the US is estimated at one in 100,000 to 200,000 live births, with most affected patients classified as severe cases.
Rocket CEO Gaurav Shah said the approval marks an important milestone for patients and families affected by the disease.
“We look forward to making KRESLADI available to eligible patients in the United States,” Shah said.
Jefferies analysts noted that while they expect KRESLADI to generate a relatively modest revenue opportunity of under $50 million, the FDA approval helps reduce risks across Rocket’s gene therapy platform and gives management valuable experience for future product launches.
They added that the Rare Pediatric Disease Priority Review Voucher could support a potential stock move of more than 20% and provide funding for the company’s AAV gene therapy pipeline.
The analysts also said Rocket’s broader AAV-based programs for rare cardiovascular diseases are positioned to advance in 2026 to 2027, pointing to the upcoming resumption of pivotal Phase II dosing for RP-A501 in Danon disease, as well as ongoing work on RP-A601 and RP-A701 in cardiomyopathy indications.
They highlighted that a PRV sale could bring in non-dilutive capital to support these programs and that KRESLADI’s US launch, though limited in scale, offers experience in payor negotiations and commercial scaling for future therapies.
After initially rising on the announcement, shares of Rocket Pharmaceuticals fell nearly 10% in early trading following the announcement.