Rocket's Kresladi gene therapy wins FDA approval for ultra-rare LAD-1
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.