Rocket Pharma jumps as gene therapy undergoes FDA review again

Rocket Pharmaceuticals (NASDAQ:RCKT) added ~11% in the premarket on Tuesday after the company announced that the FDA had accepted its resubmitted marketing application for its gene therapy candidate Kresladi.

In June 2024, the FDA rejected the company’s biologics license application for the lentiviral vector gene therapy, targeted at a rare immune disease called leukocyte adhesion deficiency-I (LAD-I). In a so-called complete response letter, the agency has requested additional data to complete the review, the company said at the time.  

In accepting the resubmitted BLA for Kresladi, the regulator has issued March 28, 2026, as the target action date for its review.

The marketing application is supported by data from a Phase 1/2 global study for the candidate in LAD-I, which reached all primary and secondary endpoints, indicating 100% overall survival for all trial subjects at 12 months.