FDA Rejects Rocket Pharmaceuticals’ Gene Therapy for Ultra-Rare Immune Disease, Requests Additional Manufacturing Information

1. FDA Rejection: The FDA rejected Rocket Pharmaceuticals' gene therapy for leukocyte adhesion deficiency-I (LAD-I), a rare and sometimes fatal immune disorder, citing the need for additional manufacturing information.
2. Complete Response Letter: The FDA issued a Complete Response Letter requesting limited additional chemistry, manufacturing, and controls (CMC) information.
3. Manufacturing Concerns: The FDA's rejection is primarily due to concerns over the manufacturing process, which is a common challenge for gene therapies.
4. Company Response: Rocket Pharmaceuticals remains committed to getting the treatment approved and has already met with the FDA to align on the limited scope of the additional information required.
5. Impact on Gene Therapy Class: This rejection is another setback for a class of gene therapies that have shown promise for rare blood and immune disorders but have proven difficult to manufacture and scale.