Rocket Pharma rises after FDA approves gene therapy for fatal childhood disorder

Rocket Pharmaceuticals

27/03/2026 11:52

** Shares of drug developer Rocket Pharmaceuticals RCKT.O rise 12.58% to $5.28 premarket

** Co says the U.S. Food and Drug Administration approved its one-time gene therapy, Kresladi, for a rare immune disorder in children

** Co says the treatment is the first FDA-approved therapy for children with this condition

** The drug aims to treat severe Leukocyte Adhesion Deficiency-I, a disorder that leaves patients highly prone to serious infections and a high risk of death in early childhood without treatment

** The FDA granted accelerated approval based on a surrogate endpoint measuring improved immune cell function

   ** The approval was backed by an early-to-mid-stage trial showing 100% survival at 12 months, no treatment-related serious side effects, and a sharp drop in severe infections

** RCKT shares were down over 72% in 2025

 (Reporting by Kunal Das in Bengaluru)

 ((Kunal.Das2@thomsonreuters.com;))