FDA Approves Rocket Pharma’s First Gene Therapy for Rare Immune Disorder

The U.S. Food and Drug Administration (FDA) has granted accelerated approval for Rocket Pharmaceuticals' gene therapy, now officially named KRESLADI (marnetegragene autotemcel). This innovative therapy specifically targets severe leukocyte adhesion deficiency-I (LAD-I), a rare condition caused by biallelic variants in the ITGB2 gene. KRESLADI is intended for patients who lack an HLA-matched sibling donor for allogeneic stem cell transplantation. Following the landmark decision, Rocket Pharma (RCKT) shares surged by 10% in pre-market trading, reflecting strong investor confidence. This approval marks the first-ever regulatory milestone for this specific condition, addressing a critical unmet medical need for patients. Market analysts view this development as a major catalyst that strengthens Rocket Pharma's leadership in the biotechnology sector and the evolving gene therapy landscape.

Sources:reuters.comfda.govstatnews.compharmaphorum.cominvesting.comstocktwits.combiospace.combusinesswire.compharmaceutical-technology.comproactiveinvestors.cabiopharmadive.comseekingalpha.com