Rocket Pharmaceuticals has achieved accelerated FDA approval for its autologous hematopoietic stem cell-based gene therapy Kresladi (marnetegragene autotemcel) for treatment of children with severe leukocyte adhesion deficiency-I (LAD-I), a rare, inherited primary immunodeficiency.
Kresladi is the first approval for the New Jersey biotech, which focuses on developing gene and cell therapies for rare hematological and cardiovascular rare diseases. It should come as a relief for the company after a challenging couple of years during which Rocket had a hold placed on its Danon gene therapy trial after a patient death and received a Complete Response Letter for Kresladi citing manufacturing issues before eventually achieving accelerated approval.
“The approval of Kresladi represents an important milestone for the severe LAD-I community,” said Gaurav Shah, MD, CEO of Rocket Pharmaceuticals, in a press statement.
“This approval reflects the dedication of patients, families, investigators, and regulators who have worked together to advance research of this ultra-rare disease.”
LAD-I is an autosomal recessive disorder of neutrophil and leukocyte function classed as ultra-rare, as it affects around one in one million births. It is caused by mutations in the ITGB2 gene and is associated with high mortality due to poor immune function. The only treatment before Kresladi was a combination of aggressive control of infections and curative allogeneic stem‑cell transplants if a suitable donor could be found.
Treatment with Kresladi is indicated for children with LAD-I without a suitable stem cell donor and involves extraction of CD34+ hematopoietic stem cells from the patient. These are then genetically modified outside the body with a self‑inactivating lentiviral vector carrying a functional ITGB2 gene. After myeloablative or intensive conditioning, a single intravenous dose of the gene‑modified cells is infused back into the patient, which should provide life‑long correction of their disease.
Accelerated FDA approval means the therapy can be marketed and sold earlier than usual but is normally contingent on pre-agreed follow up trials to confirm clinical benefit, which Rocket will have to complete. It also gives the company a Rare Pediatric Disease Priority Review Voucher due to the serious and rare nature of LAD-I, which the company can use to speed up the review of another therapeutic candidate or sell to another company to boost its capital. Rocket has stated it plans to sell the voucher to boost capital.
The approval of Kresladi is unlikely to bring in large profits for the company due to the rarity of the disease but is undoubtedly a win for Rocket after a difficult year in the gene therapy space.
