FDA approves Rocket gene therapy for rare immune disorder
statnews.com • • business
Key Points:
- The FDA approved Kresladi, a gene therapy developed by Rocket Pharma for severe leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease that makes children prone to life-threatening infections.
- Kresladi had initially been rejected by the FDA in 2024 due to manufacturing concerns but has now received approval.
- The therapy is a one-time treatment likely to cost millions, though it is not expected to generate significant revenue given the rarity of LAD-1, which affects about one in a million people.
- Kresladi is approved only for children without a matched sibling donor for stem cell transplant, the current standard treatment for LAD-1.
