FDA Rejects Rocket Pharma's Gene Therapy, Stock Falls

The US Food and Drug Administration (FDA) has declined to approve Rocket Pharmaceuticals' gene therapy, Kresladi, intended for treating Leukocyte Adhesion Deficiency-I (LAD-I), a rare and severe pediatric disorder that causes the immune system to malfunction. The FDA requested additional information regarding certain processes related to the gene therapy to complete its review. Rocket Pharmaceuticals was seeking approval to use Kresladi for patients with severe LAD-I, who suffer from a mutation in the protein-encoding ITGB2 gene, leading to severe immune system deficiencies and recurrent infections.

Following the FDA's decision, Rocket Pharmaceuticals experienced a 14% drop in premarket trading. The company had previously been granted priority review for Kresladi's biologics license application in 2023. Rocket Pharmaceuticals' CEO, Gaurav Shah, acknowledged the FDA's understanding of the high unmet medical need and the importance of timely patient access to the therapy.