Rocket Pharma shares rise on FDA approval for gene therapy
The FDA approved Kresladi for severe Leukocyte Adhesion Deficiency-I, marking the first treatment for the disorder. Shares rose 10% following the announcement.
Shares of Rocket Pharmaceuticals, Inc. surged 10% in pre-market trading on Friday following a landmark regulatory decision in the United States. The Food and Drug Administration (FDA) has approved Kresladi, a gene therapy for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I), marking the first time a therapy has been authorized specifically for this rare and often fatal immune disorder.
LAD-I is a condition that prevents white blood cells from reaching infection sites, leaving patients highly susceptible to life-threatening infections. Without a stem cell transplant, approximately 75% of untreated patients do not survive past the age of two. Kresladi is a one-time treatment that works by harvesting a patient’s own blood stem cells, genetically correcting the defective gene, and infusing the modified cells back into the body.
