STAT+: FDA rejects Rocket Pharmaceuticals’ gene therapy for ultra-rare condition, another setback for field
Rocket Pharmaceuticals said Friday that the Food and Drug Administration rejected its gene therapy for an ultra-rare, sometimes fatal immuno-deficiency syndrome, saying the company needed to submit more information to prove it can safely manufacture the product.
The New York-based biotech said the FDA asked for only “limited” additional information. A spokeswoman added the company remains “deeply committed to getting this treatment approved.”
The rejection is another setback for a class of gene therapies that have held tremendous promise for patients with rare blood and immune disorders but have proven exceptionally difficult to manufacture and scale.
