AI & Machine Learning in Biotechnology
STAT+: FDA approves Rocket gene therapy for rare immune disorder
The Food and Drug Administration approved on Thursday a gene therapy for severe leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease that leaves children vulnerable to life-threatening infections.
The therapy, marketed as Kresladi, was developed by Rocket Pharma. It was initially rejected by the FDA in 2024 over manufacturing concerns.
Though Rocket is likely to charge millions of dollars for the one-time treatment, Kresladi is not expected to be a major money maker. LAD-1 is thought to affect only around 1 in a million people, with Rocket estimating around 25 new cases per year. The treatment is approved only for children who don’t have a matched sibling who can give a stem cell transplant, the standard of care in the disease.
The Food and Drug Administration approved on Thursday a gene therapy for severe leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease that leaves children vulnerable to life-threatening infections.
The therapy, marketed as Kresladi, was developed by Rocket Pharma. It was initially rejected by the FDA in 2024 over manufacturing concerns.
Though Rocket is likely to charge millions of dollars for the one-time treatment, Kresladi is not expected to be a major money maker. LAD-1 is thought to affect only around 1 in a million people, with Rocket estimating around 25 new cases per year. The treatment is approved only for children who don’t have a matched sibling who can give a stem cell transplant, the standard of care in the disease.