Rocket Pharma moved swiftly on Monday to build on its historic FDA approval of Kresladi, the first gene therapy cleared for pediatric patients with severe leukocyte adhesion deficiency type I (LAD-I) who lack a matched sibling donor. Company executives confirmed that the FDA has agreed to review supplemental data covering adolescent patients aged 13 to 17, a cohort excluded from the original indication announced Saturday.
The approval, which landed late on Friday and generated widespread coverage over the weekend, marked a turning point for a condition that has historically carried a mortality rate exceeding 60 percent in its severe form within the first two years of life. Rocket Pharma's chief medical officer said on a Monday morning investor call that the company had anticipated the label expansion discussion and submitted a supplemental biologics license application package in February, ahead of the primary approval.
Patient advocacy groups, including the Immune Deficiency Foundation, welcomed the development and called on the Centers for Medicare and Medicaid Services to begin reimbursement pathway discussions immediately. The therapy, delivered via a lentiviral vector to correct the CD18 gene mutation underlying LAD-I, has shown durable engraftment in clinical trials with a median follow-up of 24 months.
Analysts at Jefferies and Canaccord Genuity revised their 2026 revenue projections for Kresladi upward on Monday morning, citing an estimated addressable population of approximately 200 to 250 newly diagnosed patients per year in the United States and Europe combined. The rare-disease pricing model is expected to place Kresladi among the highest-cost single-administration therapies on the market, likely in the range of $3 million to $4 million per patient.
Children's hospitals with established bone marrow transplant programs, including Boston Children's Hospital and Cincinnati Children's, confirmed they are in the credentialing process to become authorized treatment centers. Rocket Pharma said it expects five to eight certified sites to be operational in the United States by the end of the second quarter, with European Medicines Agency review of the marketing authorisation application ongoing.