Rocket's Kresladi gene therapy wins FDA approval for ultra-rare LAD-1
TL;DR Summary
The FDA approved Rocket Pharma’s one-time gene therapy Kresladi to treat severe LAD-1 in children without a matched sibling donor, following a 2024 manufacturing-based rejection. LAD-1 is ultra-rare (about 1 in a million), with roughly 25 new cases per year, and the therapy is expected to carry a multimillion-dollar price tag but is unlikely to become a major moneymaker.
- FDA approves Rocket gene therapy for rare immune disorder statnews.com
- First therapy funded by California taxpayer dollars is approved to treat rare childhood disorder GlobeNewswire
- FDA Approves First Gene Therapy for Severe LAD-1 Medscape
- Rocket charts measured trajectory for new gene therapy Kresladi after clearance to launch from FDA Fierce Pharma
- FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder UCLA Health
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