Overview
- Rocket Pharmaceuticals, which won FDA accelerated approval Friday for Kresladi to treat children with severe LAD-I, saw its stock jump as much as 9% before the open and hold about 6% higher at the start of trading.
- The therapy is indicated for pediatric patients with severe LAD-I caused by biallelic ITGB2 variants who lack a matched sibling donor for a stem cell transplant, which is the current standard of care.
- Kresladi uses a patient’s own stem cells that are engineered to add working copies of ITGB2, aiming to restore the CD18/CD11a proteins that help white blood cells stick to blood vessel walls and reach infections.
- The FDA based the accelerated decision on Phase I/II data showing higher neutrophil CD18 and CD11a levels through 12 months with durability to 24 months, and it requires longer-term follow-up and a post-approval registry to verify clinical benefit.
- Alongside approval, the FDA granted a Rare Pediatric Disease Priority Review Voucher that analysts say can sell for about $200 million, and Rocket said it will explore monetizing it after a review path that included a June 2024 rejection and an October 2024 resubmission.