US EditionRocket Pharma shares climb as FDA approves first therapy for deadly ...
- On Thursday, the U.S. Food and Drug Administration granted accelerated approval to Rocket Pharmaceuticals' gene therapy KRESLADI for pediatric patients with severe leukocyte adhesion deficiency-I lacking a matched sibling donor.
- Severe LAD-I is an ultra-rare genetic disorder caused by mutations in the ITGB2 gene that leaves children vulnerable to life-threatening infections; about 75% of untreated patients die before age two.
- Clinical trial results showed 100% survival at 12 months after infusion without treatment-related serious side effects, while KRESLADI sharply reduced serious infections in study participants.
- Rocket Pharmaceuticals shares jumped 10% on Friday following the approval, which also earned the company a Rare Pediatric Disease Priority Review Voucher worth $200 million.
- Despite previous skepticism toward surrogate endpoints, Vinay Prasad, the FDA's head of biologics, stated the agency "continues to exercise significant regulatory flexibilities" during rare-disease therapy reviews.
23 Articles
23 Articles
Medical Xpress
NewswiseFDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder
Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug Administration–approved therapy for severe leukocyte adhesion deficiency-I—a genetic condition characterized by recurrent infections and, often, early death.
Public Research 'Made in Spain' for Rare Diseases: u.s. Approves the First Spanish Gene Therapy Drug
Three years after treatment, all 9 patients treated for LAD-I (a rare severe pediatric immunodeficiency) are still alive and free from recurrent episodes of infections that may be fatal to them.
First therapy funded by California taxpayer dollars is approved to treat rare childhood disorder
South San Francisco, Calif., March 27, 2026 (GLOBE NEWSWIRE) -- The California Institute for Regenerative Medicine (CIRM) celebrates a major milestone in the agency's 21-year history: the first approved therapy to come out of California taxpayer funding. The U.S. Food and Drug Administration (FDA) has given accelerated approval to KRESLADI™, a one-time therapy developed by Rocket Pharmaceuticals, Inc. to treat severe leukocyte adhesion deficienc…
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